COVER EVENT AT A GLANCE PLENARY KEYNOTE SESSION Eighth Annual SHORT COURSES TRAINING SEMINARS THE SUMMIT UPSTREAM PROCESSING BIOPROCESSING Optimizing Cell Culture Technology Bioproduction: Scale, Bioreactors Practical Solutions for Today’s Bioprocess Challenges & Disposables Optimizing Cell Line Development DOWNSTREAM PROCESSING Continuous Processing in August 15-19, 2016 | Westin Boston Waterfront , Boston, MA Biopharm Manufacturing Advances in Purification Technologies Virus & Pathogen Clearance & Safety in Biologics Preparing for Next-Generation ANALYTICAL & QUALITY Host Cell Proteins: Detection, Products: Emerging Technologies Analysis & Removal Early Analytical Development for Biotherapeutics & Innovative Strategies Process Characterization, Qualification & Control FORMULATION & STABILITY Rapid Methods to Assess Quality & Stability of Biologics Overcoming Formulation Challenges High-Concentration Protein Formulations CELL & GENE THERAPY PRODUCTION Cell Therapy CMC, Quality & Analytics Cell Therapy Bioproduction, Operations & Logistics Gene Therapy Bioproduction Manufacturing & CMC Strategies for Biologics SPONSORS & EXHIBITORS BioprocessingSummit.com HOTEL & TRAVEL REGISTRATION INFORMATION Premier Sponsors
BioprocessingSummit.com Cambridge Healthtech Institute COVER EVENT AT A GLANCE Event at a Glance PLENARY KEYNOTE SESSION EVENT AT A GLANCE SHORT COURSES TRAINING SEMINARS Monday - Tuesday Wednesday - Thursday AM Thursday PM - Friday August 15 - 16 August 17 - 18 August 18 - 19 UPSTREAM PROCESSING STREAM #1 Optimizing Cell Culture Technology Optimizing Cell Culture Bioproduction: Scale, Bioreactors Optimizing Cell Line Technology & Disposables Development Bioproduction: Scale, Bioreactors Upstream Processing & Disposables STREAM #2 Optimizing Cell Line Development Continuous Processing in Advances in Purification Virus & Pathogen Clearance Downstream Processing Biopharm Manufacturing Technologies & Safety in Biologics DOWNSTREAM PROCESSING
Continuous Processing in STREAM #3 Host Cell Proteins: Detection, Early Analytical Development for Process Characterization, Biopharm Manufacturing Qualification & Control Analytical & Quality Analysis & Removal Biotherapeutics Advances in Purification Technologies STREAM #4 Rapid Methods to Assess Overcoming Formulation High-Concentration Virus & Pathogen Clearance Formulation & Stability Quality & Stability of Biologics Challenges Protein Formulations & Safety in Biologics
STREAM #5 ANALYTICAL & QUALITY Cell Therapy CMC, Cell Therapy Bioproduction, Gene Therapy Bioproduction Cell & Gene Quality & Analytics Operations & Logistics Host Cell Proteins: Detection, Therapy Production Analysis & Removal Manufacturing & CMC Early Analytical Development for Strategies for Biologics
Biotherapeutics Introduction to Bioprocessing Current & Emerging Global Regulatory Introduction to Analytical Method Process Characterization, Expectations for Analytical Elements of Development & Validation for Qualification & Control Introduction to Extractables Biotechnology/Biosimilar Products Therapeutic Proteins & Leachables & Packaging Introduction to Cell Culture Introduction to Downstream FORMULATION & STABILITY Identifying & Managing Risk during Processing Rapid Methods to Assess Development, Scale-Up, & Validation of Biological Processes Quality & Stability of Biologics Overcoming Formulation Cambridge Healthtech Challenges SHORT COURSES DINNER SHORT COURSES DINNER SHORT COURSES Monday, August 15 Tuesday, August 16 Thursday, August 18 High-Concentration Protein Formulations CELL & GENE THERAPY PRODUCTION Cell Therapy CMC, Quality & Analytics ABOUT THE SUMMIT Cell Therapy Bioproduction, Operations & Logistics The Bioprocessing Summit convenes more than 1,000 international bioprocess professionals to share practical solutions for today's bioprocess Gene Therapy Bioproduction challenges. Now in its eighth year, the event features 16 distinct meetings ranging from cell line development and ensuring quality, to formulation, analytical development, continuous processing, purification, viral clearance and safety, and bioproduction. New programming in Manufacturing & CMC Strategies for Biologics 2016 will continue to expand the scope of the event and includes weeklong programming on cell and gene therapy production, and a meeting on manufacturing and CMC strategies for biologics. Along with the impressive array of conferences, the Summit also includes Short Courses SPONSORS & EXHIBITORS and Training Seminars that provide in-depth coverage of critical bioprocess topics. HOTEL & TRAVEL REGISTRATION INFORMATION Give yourself a Bioprocess Holiday this summer! Stimulate and enrich yourself at the 8th Annual The Bioprocessing Summit—the leading bioprocess event! BioprocessingSummit.com ◄ 2 ► COVER EVENT AT A GLANCE PLENARY KEYNOTE SESSION PLENARY KEYNOTE SESSION Wednesday, August 17 SHORT COURSES Preparing for Next-Generation Products: Emerging Technologies & Innovative Strategies TRAINING SEMINARS UPSTREAM PROCESSING Optimizing Cell Culture Technology 4:45 Chairperson’s Remarks portfolio, directing the development of innovative manufacturing platforms and process improvements for clinical and commercial Bioproduction: Scale, Bioreactors John Sterling, Editor-in-Chief, Genetic projects. Morrey holds a B.S. from Indiana University and a Ph.D. & Disposables Engineering & Biotechnology News (GEN) from Stanford University. Prior to BMS, Morrey’s career has Optimizing Cell Line Development included various management roles at Eli Lilly and Company, Cook DOWNSTREAM PROCESSING Pharmica and Targeted Genetics Corporation. Continuous Processing in 5:25 Next-Generation Processes, Technologies and Biopharm Manufacturing 4:50 Challenges Associated With Biomanufacturing Operations Advances in Purification of Evolving Portfolios Technologies Craig Beasley, Vice President, Next Generation Morrey Atkinson, Ph.D., Vice President, Process Manufacturing, Biogen Virus & Pathogen Clearance Sciences, Bristol-Myers Squibb A critical step in meeting the demand of biologic & Safety in Biologics The accelerating pace of biological production worldwide involves implementing ANALYTICAL & QUALITY product development and high rate of disruptive manufacturing technologies, Host Cell Proteins: Detection, technology advancement is causing shifts processes and capabilities. This talk will evaluate Biogen’s Analysis & Removal in biomanufacturing. As an industry, we are new manufacturing site in Switzerland, due to go online in Early Analytical Development for being compelled to match this change with increased 2019, including the new processes, operational models and Biotherapeutics flexibility. From clinical supplies to late life-cycle commercial technologies being adopted to drive value through innovation Process Characterization, production, demand for innovation and creativity has never and deliver new medicines in areas such as Alzheimer’s. Qualification & Control been greater, particularly to reliably supply transformational 6:00 Networking Reception in the Exhibit Hall with FORMULATION & STABILITY medicines such as the new immuno-oncology products. Poster Viewing Rapid Methods to Assess E. Morrey Atkinson, Ph.D., is currently the Vice President of Quality & Stability of Biologics Biologics Development at Bristol-Myers Squibb Company. Morrey Overcoming Formulation is responsible for leading a team of scientists and engineers across CONNECT WITH US Challenges five sites that is responsible for process development, analytical sciences, and manufacturing support for the company’s biologics High-Concentration #BPSMT Protein Formulations CELL & GENE THERAPY PRODUCTION Cell Therapy CMC, Quality & Analytics Present a Poster & Save Cell Therapy Bioproduction, Operations & Logistics Cambridge Healthtech Institute encourages attendees to gain further exposure Gene Therapy Bioproduction by presenting their work in the poster sessions. To secure a poster board and Manufacturing & CMC inclusion in the conference materials, your abstract must be submitted, approved Strategies for Biologics and your registration paid in full by July 15, 2016. SPONSORS & EXHIBITORS • Your research will be seen by leaders • Your poster abstract will be published HOTEL & TRAVEL from top pharmaceutical, biotech, in the conference materials #BPSMT REGISTRATION INFORMATION academic and government institutes • Receive $50 off your registration fee BioprocessingSummit.com ◄ 3 ► COVER EVENT AT A GLANCE PLENARY KEYNOTE SESSION PREMIER SPONSOR SHORT COURSES TRAINING SEMINARS UPSTREAM PROCESSING Optimizing Cell Culture Technology Bioproduction: Scale, Bioreactors & Disposables Optimizing Cell Line Development DOWNSTREAM PROCESSING Continuous Processing in Biopharm Manufacturing Advances in Purification CORPORATE SPONSORS Technologies Virus & Pathogen Clearance & Safety in Biologics ANALYTICAL & QUALITY Host Cell Proteins: Detection, Analysis & Removal Early Analytical Development for Biotherapeutics Process Characterization, Qualification & Control FORMULATION & STABILITY Rapid Methods to Assess Quality & Stability of Biologics Overcoming Formulation Challenges High-Concentration Protein Formulations CELL & GENE THERAPY PRODUCTION Cell Therapy CMC, Quality & Analytics Cell Therapy Bioproduction, Operations & Logistics CORPORATE SUPPORT SPONSORS Gene Therapy Bioproduction Manufacturing & CMC Strategies for Biologics SPONSORS & EXHIBITORS HOTEL & TRAVEL REGISTRATION INFORMATION
BioprocessingSummit.com ◄ 4 ► COVER EVENT AT A GLANCE PLENARY KEYNOTE SESSION SHORT COURSES SHORT COURSES TRAINING SEMINARS MONDAY, AUGUST 15 TUESDAY, AUGUST 16 UPSTREAM PROCESSING MORNING COURSES | 9:00 – 11:30 AM DINNER COURSES | 6:00 – 8:30 PM Optimizing Cell Culture Technology SC1: Optimizing Media for Culturing Cells SC5: Analytical Strategies for Comparability in Bioprocess Bioproduction: Scale, Bioreactors This course will provide helpful strategies for improving process robustness, Development & Disposables extending culture viability, and increasing productivity. The course will cover Bioprocess changes can impact quality attributes of biologics and may affect Optimizing Cell Line Development essential ingredients needed to culture cells, as well as new ways to replace efficacy and safety of the product. During development and throughout traditional elements with chemically-defined components, and provide optimal the product lifecycle, when process improvements are implemented, it is DOWNSTREAM PROCESSING culture conditions. Reducing process variability and influencing key product essential to gather sufficient data to support the conclusion that product Continuous Processing in quality attributes will also be addressed. safety and efficacy has not been adversely affected. This demonstration Biopharm Manufacturing Instructors: exercise requires careful planning of the comparability studies and is based on Sara Gall, MS, Senior Engineer, Process Development, Drug Substance the background knowledge of protein structure, biological function, and clinical Advances in Purification Technology & Engineering, Amgen, Inc. attribute profiles of the product accumulated during development. Technologies Kamal A. Rashid, Ph.D., Director, Biomanufacturing Education & Training In this short course, we will discuss the key concepts of establishing Virus & Pathogen Clearance Center, Worcester Polytechnic Institute product quality profiles, defining control strategies, the common analytical characterization technologies used, and the hierarchical assessment approach & Safety in Biologics Andrew Salazar, M.Sc., Ph.D. Student, Biomaterial and Technology Development, Merck Lifescience to demonstrating comparability of the product in support of process changes. ANALYTICAL & QUALITY Catherine Lynes, MSc., MEng., Associate Scientist II, Technical Development, Instructor: Host Cell Proteins: Detection, Biogen Christine P. Chan, Ph.D., Principal Scientist/Technical Lead, Global Analysis & Removal Manufacturing Science & Technology, Specialty Care Operations, Sanofi SC2: Accelerated Stability Testing of Biologics Early Analytical Development for This short course will aim to guide the researcher in designing studies for SC7: Protein Aggregation: Mechanism, Characterization and Biotherapeutics accelerated stability testing of biologics. The course will begin with basic Consequences Process Characterization, underlying concepts governing protein drug product stability, and focus on Protein aggregation is recognized by regulatory agencies and the biopharmaceutical industry as a key quality attribute of biotherapeutics. Qualification & Control design principles for measuring stress and accelerated stability testing of not only the protein of interest, but also of excipients and primary packaging Various aggregates hold the potential for adversely impacting production FORMULATION & STABILITY components. Strategies to handle complexities arising from their interactions and patients in a variety of ways. This in-depth workshop reviews the origins will also be discussed. and consequences of aggregation in biotherapeutics, and then examines Rapid Methods to Assess strategies for predicting and quantifying aggregation in biopharmaceuticals. Quality & Stability of Biologics Instructor: Jan Jezek, Ph.D., CSO, Development, Arecor Ltd. It benefits scientists engaged in development, production, analytical characterization and approval of biotherapeutics and who require a good Overcoming Formulation Sanket Patke, Ph.D., Research Investigator, Drug Product Science and working knowledge of protein aggregation. Challenges Technology, Pharmaceutical Development, Bristol-Myers Squibb Instructor: High-Concentration SC3: New USP Initiatives and Standards for Biologics Thomas Laue, Ph.D., Professor, Biochemistry and Molecular Biology; Director, Protein Formulations • Latest updates on USP Chapters and Monographs Biomolecular Interaction Technologies Center (BITC), University of New Hampshire CELL & GENE THERAPY PRODUCTION • Feedback and comments from industry Cell Therapy CMC, • USP approaches to biologics Quality & Analytics Instructor: Cell Therapy Bioproduction, Maura Kibbey, Ph.D., Director, Science and Standards, Global Biologics, USP Operations & Logistics SC4: Advanced Process Control Strategies in Bioprocessing Gene Therapy Bioproduction This course is focused on critical issues during implementation of advanced process control strategies in bioprocesses. We will summarize specific Manufacturing & CMC aspects from data acquisition, data processing to advanced control strategies. Strategies for Biologics We will also discuss software requirements for data management and process control within and along different process scales. The topic is strongly SPONSORS & EXHIBITORS related to PAT/QbD implementation in bioprocesses. HOTEL & TRAVEL Instructor: REGISTRATION INFORMATION Gerald Striedner, Ph.D., Assistant Professor, Department of Biotechnology, University of Natural Resources and Life Sciences, Vienna, Austria, Head of Working Group Fermentation Technology, Co-Founder of enGenes Biotech BioprocessingSummit.com GmbH ◄ 5 ► COVER EVENT AT A GLANCE PLENARY KEYNOTE SESSION SHORT COURSES SHORT COURSES TUESDAY, AUGUST 16 THURSDAY, AUGUST 18 TRAINING SEMINARS DINNER COURSES | 6:00 - 8:30 PM CONTINUED DINNER COURSES | 6:00 – 8:30 PM UPSTREAM PROCESSING Optimizing Cell Culture Technology SC8: Purification of Advanced Medicine Therapy Products SC10: Transient Protein Production in Mammalian Cells Advanced Therapy Medicinal Products (ATMPs), as stem cell-based products, This short course introduces both the fundamental concepts and technologies Bioproduction: Scale, Bioreactors virus and virus-like particles have a unique complexity that challenges needed to establish transient protein production in mammalian cells. This & Disposables the design of robust and effective biomanufacturing processes. Already allows for the rapid generation, purification and characterization of milligram- Optimizing Cell Line Development established downstream processing steps should be rethought and to-gram quantities of secreted or intracellular recombinant proteins for redesigned to cope with the specific demands for these types of therapeutic therapeutic, functional and structural studies. The course combines instruction DOWNSTREAM PROCESSING products, maintaining their final quality attributes in terms of identity, potency, and case studies in an interactive environment. Continuous Processing in purity and safety. Instructors: Biopharm Manufacturing In this short course we will provide a comprehensive and detailed outline Richard Altman, MS, Scientist, Protein Technologies, Amgen, Inc. of hands-on issues for ATMPs´ purification and an overview of the main Advances in Purification Henry C. Chiou, Ph.D., Associate Director, Cell Biology, Life Science Solutions, challenges that these type of products pose and how we can tailor current Thermo Fisher Scientific Technologies processes to fit their particular requirements. Dominic Esposito, Ph.D., Director, Protein Expression Laboratory, Frederick Virus & Pathogen Clearance Instructors: National Laboratory for Cancer Research, Leidos Biomedical Research, Inc. & Safety in Biologics Ricardo Silva, Ph.D., Researcher, Downstream Processing Lab, Animal Cell Technology, iBET – Instituto de Biologia Experimental e Tecnologica ANALYTICAL & QUALITY Barbara Cunha, MSc, Animal Cell Technology, iBET – Instituto de Biologia Host Cell Proteins: Detection, Experimental e Tecnologica Analysis & Removal Early Analytical Development for Biotherapeutics Process Characterization, Qualification & Control FORMULATION & STABILITY Rapid Methods to Assess Quality & Stability of Biologics Overcoming Formulation Challenges High-Concentration Protein Formulations CELL & GENE THERAPY PRODUCTION Cell Therapy CMC, Quality & Analytics Cell Therapy Bioproduction, Operations & Logistics Gene Therapy Bioproduction Manufacturing & CMC Strategies for Biologics SPONSORS & EXHIBITORS HOTEL & TRAVEL REGISTRATION INFORMATION
BioprocessingSummit.com ◄ 6 ► COVER Cambridge Healthtech EVENT AT A GLANCE PLENARY KEYNOTE SESSION SHORT COURSES MONDAY, AUGUST 15 - TUESDAY, AUGUST 16 TRAINING SEMINARS DAY 1 1:00-5:00 PM • DAY 2 8:00 AM-5:15 PM UPSTREAM PROCESSING Optimizing Cell Culture Technology (TS1) Introduction to Bioprocessing (TS3) Identifying and Managing Risk during Development, Scale- The Introduction to Bioprocessing training seminar offers a comprehensive Up, and Validation of Biological Processes Bioproduction: Scale, Bioreactors survey of the steps needed to produce today’s complex biopharmaceuticals, This seminar will look at the common, and some less common tools for risk & Disposables from early development through commercial. We begin with a brief introduction assessment and risk management and will provide guidance on how these Optimizing Cell Line Development to biologic drugs and the aspects of protein science that drive the intricate tools can be applied to the management of risk in pharmaceutical development, progression of analytical and process steps that follows. We then step through process scale-up and process validation of biological processes. The seminar will DOWNSTREAM PROCESSING the stages of bioprocessing, beginning with the development of cell lines and consist of presentations, break out workshops and discussions of examples of Continuous Processing in ending at scaling up for commercial production. The seminar also explores successful risk management programs. emerging process technologies, facility design considerations and regulatory Biopharm Manufacturing • Historical and regulatory background of risk management; mitigating and and quality standards. managing risk Advances in Purification • Introduction to biopharmaceuticals and bioprocessing • Identification of risks in process development, scale-up and Technologies • Analytical methods for characterization and release process manufacturing Virus & Pathogen Clearance • Quality systems • Tools for risk assessment in pharmaceutical development and manufacturing & Safety in Biologics • The science and technologies of bioprocess unit operations • Examples of the application of risk assessment tools in development, scale- ANALYTICAL & QUALITY • Drug product development up and process validation • New approaches to validation of bioprocesses Host Cell Proteins: Detection, Instructors: Analysis & Removal Instructor: Trevor Deeks, Ph.D., QA and QC Consultant, Teva Biopharmaceuticals Early Analytical Development for Sheila G. Magil, Ph.D., Senior Consultant, BioProcess Technology USA, Inc. Biotherapeutics Consultants, Inc. Process Characterization, Qualification & Control Frank J. Riske, Ph.D., Senior Consultant, BioProcess Technology FORMULATION & STABILITY Consultants, Inc. Rapid Methods to Assess (TS2) Introduction to Extractables and Leachables and Packaging Quality & Stability of Biologics Chemical substances can be leached into biologics from various components Overcoming Formulation used in the manufacture, storage or delivery of a therapeutic product leading to a negative impact on the product and potential for an undesirable effect Challenges on the patient. This training seminar will provide a background on regulatory High-Concentration expectations for materials and components in contact with biologics and the Protein Formulations unique applications to biologic delivery systems. • Regulatory expectations for container closure and delivery systems CELL & GENE THERAPY PRODUCTION • Overview of materials used to manufacture, store and deliver biologics Cell Therapy CMC, • Designing studies to qualify materials for intended use Quality & Analytics • Controlled extractables testing (CES) Cell Therapy Bioproduction, • Leachables studies Operations & Logistics Gene Therapy Bioproduction Instructors: Diane Paskiet, MS, Senior Director, Global Scientific Affairs, West Manufacturing & CMC Pharmaceuticals Strategies for Biologics SPONSORS & EXHIBITORS Fran Degrazio, Vice President, Global Scientific Affairs And Technical HOTEL & TRAVEL Services, West Pharmaceuticals REGISTRATION INFORMATION
BioprocessingSummit.com ◄ 7 ► COVER Cambridge Healthtech EVENT AT A GLANCE PLENARY KEYNOTE SESSION SHORT COURSES TRAINING SEMINARS WEDNESDAY, AUGUST 17 - THURSDAY, AUGUST 18 DAY 1 8:00 AM-3:50 PM • DAY 2 8:30 AM-12:15 PM UPSTREAM PROCESSING Optimizing Cell Culture Technology (TS5) Introduction to Cell Culture • Equipment use and decontamination Bioproduction: Scale, Bioreactors This 1.5-day Intro to Cell Culture training seminar is a lecture based course • Contamination prevention and types of contamination & Disposables intended for the beginner who is thinking about culturing animal cells for the • Cell culture media first time or for intermediate cell culturists wanting to know more about how • Clonal isolation of animal cells Optimizing Cell Line Development animal cell culture works and how to improve their process. Attendees will learn • Primary culture and animal cell attachment and signaling; growth curves, DOWNSTREAM PROCESSING about most of the critical aspects of cell culture from equipment maintenance and media selection to cell growth and cryopreservation. Participants will have strategies for growing animal cells in culture Continuous Processing in ample time to ask specific questions and get worthwhile answers. Biopharm Manufacturing Instructor: Timothy Fawcett, Ph.D., Scientific Director, BioTechnical Institute of Advances in Purification Maryland,Inc. and Founder, BioSciConcepts Technologies THURSDAY, AUGUST 18 - FRIDAY, AUGUST 19 Virus & Pathogen Clearance DAY 1 2:00-5:15 PM • DAY 2 8:30 AM-3:15 PM & Safety in Biologics ANALYTICAL & QUALITY (TS6) Introduction to Analytical Method Development and (TS7) Introduction to Downstream Processing Host Cell Proteins: Detection, Validation for Therapeutic Proteins This activity-packed course focuses on the science, technologies and strategies needed to understand and implement an effective downstream process for Analysis & Removal This course is a panoramic review of analytical method development and validation for therapeutic proteins, including antibodies and enzymes. It biological development and production. The course begins with an in-depth look Early Analytical Development for is intended for scientists working on therapeutic proteins in Analytical at DSP design and development – from recovery to purification to formulation Biotherapeutics Development, Quality Control, Product Development or related functional - before moving onto pertinent issues surrounding HTPD, single-use systems, continuous processing, PAT, CPPs, viral clearance, platform development and Process Characterization, areas. The course emphasizes practical applications, real-world examples and useful tips. process validation. The course concludes with real-world examples for both Qualification & Control • Manufacturing of protein drugs, regulatory affairs, protein chemistry traditional and emerging modalities. FORMULATION & STABILITY • Commonly used analytical methods for proteins • Intro and principles of downstream processing Rapid Methods to Assess • Method validation and method transfer • Recovery and Purification Quality & Stability of Biologics • Regulatory compliance at different stages of product development • Future considerations and challenges Overcoming Formulation • Application of DOE and QbD • Process design & economics; process validation; continuous processing Challenges • Quality by Design and critical process parameters; analytics and PAT solutions Instructor: High-Concentration Instructor: Professor Jean-Francois Hamel, Academic Researcher and Instructor, Protein Formulations Jichao (Jay) Kang, Ph.D., RAC, Director, Analytical and Formulation Development,Patheon Biologics Chemical Engineering Department, Massachusetts Institute of CELL & GENE THERAPY PRODUCTION Technology Cell Therapy CMC, Quality & Analytics Each CHI Training Seminar offers 1.5 Days of instruction with start and stop times for each day shown above and on the Event-at-a-Glance published in the onsite Program & Event Guide. Training Seminars will include morning and afternoon refreshment breaks, as applicable, and lunch will be provided to all registered Cell Therapy Bioproduction, attendees on the full day of the class. Operations & Logistics Each person registered specifically for the training seminar will be provided with a hard copy handbook for the seminar in which they are registered. A limited Gene Therapy Bioproduction number of additional handbooks will be available for other delegates who wish to attend the seminar, but after these have been distributed no additional books will be available. Manufacturing & CMC Though CHI encourages track hopping between conference programs, we ask that Training Seminars not be disturbed once they have begun. In the interest Strategies for Biologics of maintaining the highest quality learning environment for Training Seminar attendees, and because Seminars are conducted differently than conference programming, we ask that attendees commit to attending the entire program, and NOT engaging in track hopping, as to not disturb the hands-on style instruction SPONSORS & EXHIBITORS being offered to the other participants. HOTEL & TRAVEL To view Training Seminar Agendas in full and Instructor Biographies, visit: REGISTRATION INFORMATION BioprocessingSummit.com/BPD/Training-Seminars BioprocessingSummit.com ◄ 8 ► COVER EVENT AT A GLANCE PLENARY KEYNOTE SESSION STREAM #1 SHORT COURSES TRAINING SEMINARS UPSTREAM PROCESSING Upstream Processing Optimizing Cell Culture Technology Bioproduction: Scale, Bioreactors & Disposables The weeklong Upstream Processing stream spans cell line selection and developability through the creation of scale-down Optimizing Cell Line Development models and scaling up production. Along the way, developing a greater understanding of cells, particularly CHO, will be DOWNSTREAM PROCESSING Continuous Processing in examined, as well as tools and technologies that support analyzing quality to more rapidly achieve productivity and mitigate Biopharm Manufacturing risk. Bioreactors and disposable / single-use systems will also be explored in this weeklong investigation of the ‘nuts and Advances in Purification Technologies bolts’ of bioprocessing. Virus & Pathogen Clearance & Safety in Biologics ANALYTICAL & QUALITY Host Cell Proteins: Detection, Analysis & Removal Early Analytical Development for AUGUST 15-16 Biotherapeutics Process Characterization, Optimizing Cell Culture Technology Qualification & Control FORMULATION & STABILITY Rapid Methods to Assess AUGUST 17-18 Quality & Stability of Biologics Overcoming Formulation Bioproduction: Scale, Bioreactors & Disposables Challenges High-Concentration Protein Formulations AUGUST 18-19 CELL & GENE THERAPY PRODUCTION Cell Therapy CMC, Optimizing Cell Line Development Quality & Analytics Cell Therapy Bioproduction, Operations & Logistics Gene Therapy Bioproduction Manufacturing & CMC Strategies for Biologics SPONSORS & EXHIBITORS HOTEL & TRAVEL REGISTRATION INFORMATION
BioprocessingSummit.com COVER Upstream Processing EVENT AT A GLANCE 12th Annual PLENARY KEYNOTE SESSION SHORT COURSES Optimizing Cell Culture Technology TRAINING SEMINARS UPSTREAM PROCESSING Enhancing Knowledge for Growing Cells Optimizing Cell Culture Technology Bioproduction: Scale, Bioreactors MONDAY, AUGUST 15 apoptosis based on macromolecular changes in 4:00 Using High-Seeding Density to & Disposables the cell composition. Also, we are addressing the Improve Cell Culture Process Efficiency and 8:00 am Short Course Registration increasing need for high cell concentration perfusion Increase Manufacturing Facility Capacity Optimizing Cell Line Development technology, including in scale-down bioreactors. Ning Liu, Ph.D., Associate Senior Consultant 9:00 – 11:30 Recommended Morning DOWNSTREAM PROCESSING 2:15 Early Development Strategies for Engineer, Bioproduct Research and Development, Short Course* Eli Lilly and Company Continuous Processing in Innovative Therapeutic Molecules Optimizing Media A high-seeding density fed-batch process with N-1 Biopharm Manufacturing Nicola Beaucamp, Ph.D., Head, Process Research, * Separate registration required, see page 5 for details. perfusion culture was developed at Lilly that has Large Molecule Research, Pharma Research and Advances in Purification significantly increased volumetric productivity. A Early Development, Roche Innovation Center Technologies case study will be presented to demonstrate that Penzberg 11:30 Main Conference Registration Opens the new process could shorten the production time Virus & Pathogen Clearance A number of novel antibody formats have been from 14 days to 7 days and achieve the same titer & Safety in Biologics QUALITY & PRODUCTIVITY advanced into clinics by Roche pRED. In order to with comparable product qualities, or increase the discover and develop differentiated monoclonal titer by 50%. Considerations for implementation at ANALYTICAL & QUALITY 1:00 pm Chairperson’s Opening Remarks antibodies Roche’s strategy is based on engineering manufacturing sites will also be discussed. Host Cell Proteins: Detection, Richard Altman, M.S., Scientist - Protein technologies which bear several challenges for Analysis & Removal Technologies, Amgen, Inc. technical development. Examples on innovative 4:30 Breakout Discussions therapeutic molecules for multi-pathway-inhibition Early Analytical Development for 1:10 OPENING KEYNOTE and specific tumor-targeting will be given. 5:30 Grand Opening Reception in the Biotherapeutics PRESENTATION: Exhibit Hall with Poster Viewing 2:45 Refreshment Break Process Characterization, Trends and Aspects of Improving Cell 7:00 End of Day Qualification & Control Culture Process Productivities QUALITY & PRODUCTIVITY FORMULATION & STABILITY Thomas Ryll, Ph.D., Vice President, Process and Analytical Development, ImmunoGen, Inc. 3:15 Strategies for Optimizing the TUESDAY, AUGUST 16 Rapid Methods to Assess A number of driving forces have resulted Productivity of a Cell Culture Platform 7:30 am Registration Opens and Morning Quality & Stability of Biologics in enormous improvements to cell culture without Impacting Product Quality Coffee Overcoming Formulation production processes over the last 25 years. Nattu Vijayasankaran, Ph.D., Senior Engineer, Late Challenges Based on the author’s experience, titers of Stage Cell Culture, Genentech Inc. – A Member of antibody fed-batch production have doubled the Roche Group CULTURING CHO CELLS High-Concentration about every 6-7 years. This presentation Cell culture processes are generally simplified Protein Formulations will review some of the driving forces and 7:55 Chairperson’s Remarks when needed as a robust platform technology. But accomplishments, and will give a personal Jonathan Bones, Ph.D., Principal Investigator, both culture medium and process parameters are CELL & GENE THERAPY PRODUCTION opinion on what we can expect in terms of National Institute for Bioprocessing Research & customized for specific cell lines when there is a Cell Therapy CMC, productivity and process robustness in the years Training (NIBRT), University College Dublin need to achieve high titer. A case study will be used Quality & Analytics to come. to demonstrate that changes made to the platform 8:00 Using Quantitative Proteomics to Link Cell Therapy Bioproduction, 1:45 FEATURED PRESENTATION: process to maximize productivity could alter the Critical Quality Attributes to Critical Process Operations & Logistics Analytical and Device Technologies for product quality profile. Strategies to minimize these Parameters product quality changes will be discussed. Gene Therapy Bioproduction Bioprocess Engineering Jonathan Bones, Ph.D., Principal Investigator, James Piret, Sc.D., Professor, Michael Smith 3:45 Selected Poster Presentation: National Institute for Bioprocessing Research & Training (NIBRT), University College Dublin Manufacturing & CMC Laboratories, University of British Columbia Development of a CHO Cell Culture Strategies for Biologics This presentation is focused on research to improve Platform for Monoclonal Antibody Quantitative proteomics is a powerful tool to understand cellular behavior at the molecular level. SPONSORS & EXHIBITORS the understanding and performance of mammalian Production: From Clone Generation to cell based processes. We have developed single- Quantitative proteomics of an IgG1 mAb producing Pilot-Plant Scale-Up HOTEL & TRAVEL cell Raman spectroscopy methods for cell analysis CHO cell line was performed following systematic to detect the types of Chinese Hamster Ovary Julien Robitaille, Research Council Officer, Cell alteration of bioprocess conditions using a limited REGISTRATION INFORMATION cell death and to detect the early stages of Culture Scale Up, National Research Council Canada Plackett-Burman design of experiments approach. In BioprocessingSummit.com ◄ 10 ► COVER Upstream Processing EVENT AT A GLANCE 12th Annual PLENARY KEYNOTE SESSION SHORT COURSES Optimizing Cell Culture Technology TRAINING SEMINARS Enhancing Knowledge for Growing Cells UPSTREAM PROCESSING Optimizing Cell Culture Technology addition to investigating changes in cellular behavior, chemically defined feed medium was used to 11:30 Genome-Scale RNA Interference Bioproduction: Scale, Bioreactors complete characterization of the expressed mAb replace hydrosylate-containing feed in the original Screen Identifies Key Pathways and & Disposables was also performed to investigate the link between Phase I process. For better control of the nutrient Genes for Improving Recombinant Protein Optimizing Cell Line Development product critical quality attributes and alterations in levels in the bioreactor, a system developed for Production in Mammalian Cells critical process parameters. automatic bioreactor sampling and feedback control DOWNSTREAM PROCESSING was integrated, enabling sampling and feed multiple Joseph Shiloach, Ph.D., Director, Biotechnology 8:30 Harmonizing Transient and Stable CHO Core Laboratory, Intramural Research, NIDDK/NIH Continuous Processing in times a day. Expression Platforms for Early-Phase Drug A high-throughput RNA interference screen Biopharm Manufacturing Discovery 9:45 Coffee Break in the Exhibit Hall with was conducted using HEK293 cells expressing firefly luciferase. The gene encoding the ornithine Advances in Purification Yashas Rajendra, Ph.D., Research Scientist, Bio-TDR Poster Viewing decarboxylase antizyme1 was selected for Technologies Cell Culture, Eli Lilly and Company EMERGING CELL CULTURE detailed investigation, and our study shows Virus & Pathogen Clearance We describe the development of transient CHO that this gene is a novel target for improving & Safety in Biologics system capable of rapidly (7 days) generating high TECHNOLOGIES expression of recombinant proteins. The genome- titers, scalable up to 6L. Additionally, we describe scale screening demonstrated in this work can 10:30 Next-Generation Cellular Models for ANALYTICAL & QUALITY the use of stable CHO pools (instead of master establish the foundation for targeted design of Host Cell Proteins: Detection, wells or clones) for generation of gram quantities Drug Discovery an efficient mammalian cell platform for different of therapeutic protein. Using the same CHO Analysis & Removal T. Oliver Chao, Ph.D., Head, Emerging Biomedical biotechnological applications. cell line and media package for both platforms Sciences, Strategy & Innovation, Sanofi Early Analytical Development for streamlines expression during early phase biologic How to improve cell culture technology for a more 12:00 pm Use of a Scalable, Sponsored By Biotherapeutics drug discovery. relevant and more translational models for drug Continuous Processing Platform discovery? From stem cells to artificial cells, from Process Characterization, 9:00 An ‘Omics’ Approach to Understanding to Solve Production Challenges in Qualification & Control single cell platforms to organs-on-chips, from Mammalian Cell Cultures Performance of a CHO-Fc Fusion Process at cultures derived from humanized animals to 3D Scott Waginer, Vice President, Bioservices, Cell 5000-L Scale cultures built by human cells. We will discuss FORMULATION & STABILITY Culture Company Amanda M. Lewis, Ph.D., Scientist II, Biologics several emerging approaches that hopefully would Rapid Methods to Assess Traditionally difficult-to-express proteins perform Development, Bristol-Myers Squibb help us to achieve this goal. Quality & Stability of Biologics well in a continuous processing bioreactor system A CHO bioprocess, where the product is a sialylated as it provides long-term sustainable homeostatic Overcoming Formulation Fc-fusion protein, showed significant variation 11:00 Mammalian Cell Fluid Mechanics and pH conditions, metabolite and waste levels, as of sialylation at large scale. Lower sialylation Scale-Up Considerations for Cell Therapy Challenges well as continuous collection of the protein of correlated with elevated mannose, a shift in glucose Peter Amaya, Ph.D. Candidate, Graduate Research High-Concentration interest for 3-9 months. This stable, in vivo like metabolism, and increased oxidative stress. This Associate, William G. Lowrie Department of environment enables increased cell viability and Protein Formulations information was used to optimize a 5-L scale-down Chemical and Biomolecular Engineering, The Ohio a higher yield compared to typical fed-batch model able to reproduce the large-scale phenotypic CELL & GENE THERAPY PRODUCTION State University systems. This presentation will highlight the profiles. Targeted transcriptomics and metabolomics The perception of “shear sensitivity” has historically steady-state production of a monoclonal antibody Cell Therapy CMC, were used to develop a proposed biological put an arbitrary upper limit on agitation and aeration and the scalability of an automated single-use, Quality & Analytics mechanism linking large-scale operation, oxidative in bioreactor operation; however, as cell densities perfusion bioreactor. Cell Therapy Bioproduction, stress, and sialylation. and productivities continue to increase, mass transfer requirements can exceed those imposed 12:30 ExpiCHO: Latest Sponsored By Operations & Logistics 9:30 Feed Optimization by these arbitrary low limits. This presentation will Developments in High-Titer Gene Therapy Bioproduction to Improve Productivity Sponsored By mainly focus on some recent experimental data Transient CHO Expression and Product Quality Enabled on microcarrier cultures regarding the effect of Manufacturing & CMC Jonathan Zmuda, Ph.D., Director, Cell Biology, by Automated Sampling System hydrodynamic forces on industrially relevant animal Thermo Fisher Scientific Strategies for Biologics Linda Hoshan, Senior Scientist, Process cells, and on scale-up. The Gibco Expi293 and ExpiCHO Expression SPONSORS & EXHIBITORS Development & Engineering, Bioprocess Development, Merck Research Laboratories Systems have become premier tools for protein expression in many pharma, biotech and academic HOTEL & TRAVEL A study in optimizing feed media and feeding laboratories, enabling high-titer recombinant strategies for a fed-batch CHO culture producing REGISTRATION INFORMATION protein production for a broad range of research monoclonal antibody will be presented. Fully applications. Here, we present the latest updates BioprocessingSummit.com ◄ 11 ► COVER Upstream Processing EVENT AT A GLANCE 12th Annual PLENARY KEYNOTE SESSION SHORT COURSES Optimizing Cell Culture Technology TRAINING SEMINARS UPSTREAM PROCESSING Enhancing Knowledge for Growing Cells Optimizing Cell Culture Technology Bioproduction: Scale, Bioreactors on the ExpiCHO Expression System with regard have been developed. Using semi-empirical and impact of HTST-treated basal and feed media was & Disposables to optimizing protein expression, purification DOE strategies, methods and processes were evaluated for a monoclonal antibody in scale-down and characterization from 96 well plates up to developed for both systems with quality and production bioreactors. The results demonstrate Optimizing Cell Line Development 10L bioreactors. productivity targets. A titer of > 3 g/L was obtained that implementing HTST-treatment of basal and for the single-chain antibody. feed media into manufacturing for this monoclonal DOWNSTREAM PROCESSING 1:15 Dessert Refreshment Break in the antibody is feasible. Continuous Processing in Exhibit Hall with Poster Viewing 3:00 Production of Recombinant Biopharm Manufacturing Biotherapeutic Proteins in Spodoptera 4:45 Optimization, Simplification and Intensification of Cell Culture Processes Advances in Purification frugiperda (Sf9) Insect Cells for Vaccine Jochen B. Sieck, Ph.D., Lab Head, Cell Culture Technologies Development IMPROVING & INNOVATING Rajiv Gangurde, Ph.D., Associate Director, Protein Media R&D, Process Solutions R&D, Merck KGaA Virus & Pathogen Clearance PROCESSES Production, Genocea Biosciences Fed-batch processes are the standard cultivation & Safety in Biologics Therapeutic proteins used for the prevention method for the manufacturing of biopharmaceuticals 1:55 Chairperson’s Remarks or treatment of human diseases have been with CHO cells today. Besides the production ANALYTICAL & QUALITY James Piret, Ph.D., Professor, Michael Smith manufactured in a variety of different expression organism, cell culture media and feeds have a great Host Cell Proteins: Detection, Laboratories, University of British Columbia systems, with baculovirus-infected insect cells impact on process productivity optimization. Our Analysis & Removal providing a solid platform for the production of recent introduction of chemically modified amino 2:00 Strategies to Improve Process acids for cell culture media allow for a significant Early Analytical Development for glycosylated proteins. One of our lead programs, Robustness for Monoclonal Antibody GEN-003, is a therapeutic HSV-2 vaccine containing simplification of CHO fed-batch processes by Biotherapeutics Production: A Case Study two proteins expressed independently in insect eliminating the second, typically caustic feed. Furthermore, increases in productivity were Process Characterization, Jeffrey Ly, MS, Senior Scientist, Process cells. Here we discuss strategies, challenges observed, resulting in more productive fed-batch as Qualification & Control Development and Commercialization, Merck & Co., and advantages of employing this system for the well as highly intensified perfusion processes. Inc. production of GEN-003. FORMULATION & STABILITY During the manufacture of a monoclonal antibody, 3:30 Refreshment Break in the Exhibit Hall 5:15 End of Conference Rapid Methods to Assess consistent product quality was maintained, but Quality & Stability of Biologics large variations in bioreactor harvest titer (>2X) with Poster Viewing 5:15 Registration for Dinner Short Courses Overcoming Formulation were observed between lots. Investigations into this titer variation led us to identify correlations OPTIMIZING CELL CULTURE 6:00 – 8:30 Challenges between cellular metabolism, feeding strategy and CONDITIONS Recommended Dinner Short Course* High-Concentration harvest titer. As a result, process modifications Analytical Strategies for Comparability in Protein Formulations have successfully been developed to obtain a 4:15 Case Study: Evaluation of High Bioprocess Development more robust production bioreactor process while Temperature-Short Time Treatment of * Separate registration required, see page 5 for details. CELL & GENE THERAPY PRODUCTION maintaining product quality. Basal and Feed Media for Production of a Cell Therapy CMC, Monoclonal Antibody 2:30 Challenges in Cell-Line, Process and Quality & Analytics Amanda L. Carpenter, MS, Associate Scientist Analytical Development of a Single-Chain II, Drug Substance Manufacturing Sciences and Cell Therapy Bioproduction, Antibody and Solutions Operations & Logistics Technology (Upstream), Bristol-Myers Squibb Hanuman Mallubhotla, Ph.D., Research Director, In the biopharmaceutical industry, high temperature- Gene Therapy Bioproduction Biopharmaceutical Development, Syngene short time (HTST) treatment of cell culture media International, Ltd. and solutions is being implemented to mitigate Manufacturing & CMC Single chain antibodies are more difficult to express the risk of potential viral or other contamination Strategies for Biologics well than full-length antibodies, and have additional of the cell culture. As a proof-of-concept, the issues with their purification. Two clone systems SPONSORS & EXHIBITORS HOTEL & TRAVEL REGISTRATION INFORMATION
BioprocessingSummit.com ◄ 12 ► COVER Upstream Processing EVENT AT A GLANCE 6th Annual PLENARY KEYNOTE SESSION SHORT COURSES Bioproduction: Scale, Bioreactors & Disposables TRAINING SEMINARS Making it Work UPSTREAM PROCESSING Optimizing Cell Culture Technology WEDNESDAY, AUGUST 17 9:30 Multivariate Data Analysis for 11:15 The Decision Process for the Bioproduction: Scale, Bioreactors Glycoprotein Manufacturing Process Utilization of Disposables in Bioprocessing & Disposables 7:00 am Registration Opens and Morning Improvement: A Case Study of Raw Stefan Schmidt, Ph.D., MBA, Vice President, Coffee Process Science and Production, Rentschler Optimizing Cell Line Development Material Control Impact on Process Performance and Product Quality Biotechnology DOWNSTREAM PROCESSING KEEPING UP WITH Siguang Sui, Ph.D., Development Scientist II, The competition between stainless steel and Continuous Processing in PRODUCTION DEMAND Late Stage Upstream Development, Alexion single-use equipment has increased over the last decade. We have systematically assessed Biopharm Manufacturing Pharmaceuticals, Inc. 8:05 Chairperson’s Opening Remarks A glycoprotein manufacturing process exhibited out- the rationale when it makes sense to implement Advances in Purification Stefan Junne, Ph.D., Group Leader and Chair, of-trend lower sialylation in the bulk drug substance. disposable equipment and where a conventional Technologies Bioprocess Engineering, Biotechnology, Technische Multivariate statistical analysis revealed sialylation approach is favorable. Here we discuss the main Universität Berlin was correlated to many process parameters and decision points regarding cost, scale and safety and Virus & Pathogen Clearance illustrate our reasoning by a number of case studies & Safety in Biologics attributes, among which the daily viabilities from 8:15 KEYNOTE PRESENTATION: N-1 and production bioreactor, though only differed and examples. by a few percent, had the strongest correlation Sponsored By ANALYTICAL & QUALITY Innovation in Bioprocessing and 11:45 Expansion of Human observed. Small-scale experiments were conducted Host Cell Proteins: Detection, Manufacturing Facility Design – Current Stem Cells in BioBLU® Trends to evaluate the correlation of Pluronic lot on the Analysis & Removal process performance and product quality. Single-Use Vessels from Eppendorf Berthold Bödeker, Ph.D., Chief Scientist, Global Early Analytical Development for Stacey Willard, Ph.D., Senior Technical Applications Drug Discovery - Global Biologics, Biotech 10:00 Coffee Break in the Exhibit Hall with Specialist, Bioprocess, Eppendorf Biotherapeutics Development, Bayer AG Poster Viewing Experimentation involving human stem cells (HSCs) Process Characterization, Disposables, closed system operation, is one of the fastest growing fields in research and Qualification & Control and continuous processing, as well as less INTEGRATING DISPOSABLES development due, in large part, to its potential for segregated simplified plant design, have made FORMULATION & STABILITY revolutionizing human disease treatment. There are significant advances in the past years. This 10:45 Single-Use Versus Stainless multiple HSC platforms being investigated, including Rapid Methods to Assess talk will summarize several aspects of these Steel Bioreactors: Quality Factors for human mesenchymal stem cells (hMSCs) and innovative elements on modern bioprocessing Quality & Stability of Biologics Consideration when Selecting a Suitable human induced pluripotent stem cells (hiPSCs). and plant design as well as some risks System Overcoming Formulation associated with these technologies particularly in 12:00 pm Sponsored Presentation Challenges the area of continuous processing and ball room Trevor Deeks, Ph.D., QA and QC Consultant, Teva Biopharmaceuticals USA, Inc. (Opportunity Available) High-Concentration concept plant design. The relative merits of single-use as compared to Sponsored By Protein Formulations 12:15 Luncheon Presentation: 9:00 FEATURED PRESENTATION: stainless steel bioreactors has been widely covered Optimize Continuous in the literature and in conference presentations. CELL & GENE THERAPY PRODUCTION Developing and Adapting Manufacturing Processing with Smarter Tools Relative cost, convenience and capabilities have Processes for More Complex Products Cell Therapy CMC, been the main areas for discussion, but there Barbara Paldus, Ph.D., CEO, Finesse Solutions Lada Laenen-Horvat, Ph.D., Senior Director & Quality & Analytics has been relatively little debate about Quality The current biopharma development and Head, Allston Manufacturing Science & Technology, Cell Therapy Bioproduction, considerations. Quality considerations are not often manufacturing models create challenges for the Genzyme Corp., A Sanofi Company enterprise to meet their goals for high success Operations & Logistics part of the decision-making process to determine the type of system to select. This presentation rates, lower cost and faster time to market. With the Gene Therapy Bioproduction discusses these Quality factors and the relevance paradigm shifting from titer to process optimization to the process, and how these factors might and analytics, the biopharma business model Manufacturing & CMC influence the decision to use single-use or stainless can be improved using continuous processing to Strategies for Biologics steel systems. manage complexity. Smart technology exists today which can increase flexibility, decrease cost, and SPONSORS & EXHIBITORS improve production. HOTEL & TRAVEL 1:00 Session Break REGISTRATION INFORMATION
BioprocessingSummit.com ◄ 13 ► COVER Upstream Processing EVENT AT A GLANCE 6th Annual PLENARY KEYNOTE SESSION SHORT COURSES Bioproduction: Scale, Bioreactors & Disposables TRAINING SEMINARS Making it Work UPSTREAM PROCESSING strategies evolve. This case study will describe 8:30 Expression Tuning for Enhanced Optimizing Cell Culture Technology SCALING UP & DOWN the implementation of a new single-use benchtop Recombinant Protein Production in E.coli Bioproduction: Scale, Bioreactors 1:45 Chairperson’s Remarks bioreactor scale-down model to support late-phase David J. Wurm, Project Assistant, Biochemical & Disposables Trevor Deeks, Ph.D., QA and QC Consultant, Teva process characterization studies and future, flexible Engineering, Vienna University of Technology manufacturing strategies. Collaboration with Biopharmaceuticals USA, Inc. Strong induction of recombinant protein production Optimizing Cell Line Development CMO and comparability of process performance in E. coli can lead to agglomeration of inactive 1:50 Bioreactor Concepts and Soft Sensor and product quality relative to existing models DOWNSTREAM PROCESSING product. We developed a feeding strategy to tune Tools for Scale-Up and Down is discussed. Continuous Processing in recombinant protein expression on a cellular level Stefan Junne, Ph.D., Group Leader and Chair, Biopharm Manufacturing 3:20 Development and Process Verification which leads to higher yields of soluble and active Bioprocess Engineering, Biotechnology, Technische product. We successfully applied this system to the of Novel Scale-Down Tools for Periplasmic Advances in Purification Universität Berlin production of 1) GFP, 2) a pharmaceutically highly Technologies For scale-down experiments, several designs Fab Extraction relevant enzyme and 3) an antibody fragment. Asma Ahmad, Eng.D., Researcher, Biochemical Virus & Pathogen Clearance of multi- and single-use bioreactors are applied. Scalability is of basic importance to mimic industrial Engineering, University College London 9:00 Increasing Protein Production with & Safety in Biologics scale conditions properly. Aside from stirred A 20ml miniature vessel was designed and used Novel Cell Ess Supplement ANALYTICAL & QUALITY reactors, rocking and orbitally shaken single- as a scale-down model to successfully mimic the Adam Elhofy, Ph.D., CSO, Essential Pharmaceuticals use bioreactors exhibit a very good scalability. periplasmic Fab extraction process 10,000 fold Enhancing protein production is a common goal in Host Cell Proteins: Detection, Application of process analytical technologies from the 200L scale, using constant power per unit Analysis & Removal the biomanufacturing industry. At a concentration of support the identification of suitable scale down volume (P/V). The process has been scaled down a 1% Cell Ess supplement resulted in a 37% increase Early Analytical Development for conditions: whenever lack of knowledge restrict further 10 fold into disposable 24 micro-well plates in productivity. When using the supplement as a Biotherapeutics a classical engineering approach, physiological and recent data comparing 20L and 2L extractions feed it resulted in in a 25% increase in yield and an and morphological features of cells should to 2ml is promising and indicates feasibility of using extension of peak protein production. Functional Process Characterization, be considered. plates to increase throughput. protein increase and desired glycosylation achieved. Qualification & Control Our results suggest that an increase in protein 2:20 Scaling-Down Local Mixing Effects for 3:50 Refreshment Break in the Exhibit Hall FORMULATION & STABILITY production may not necessarily require a change in Biotechnology Applications with Poster Viewing the metabolic state of the cells. Rapid Methods to Assess Marcio B. Machado, Ph.D., Research Associate, Quality & Stability of Biologics Chemical and Materials Engineering, University of 4:45 Plenary Keynote Session (see page 3 9:30 Using Simulation to Boost Bioreactor for details) Overcoming Formulation Alberta and Process Equipment’s Productivity Challenges One of the most critical steps for a process 6:00 Networking Reception in the Exhibit Luke Munholand, Ph.D., Lead Engineer, ANSYS, Inc scale-down or scale-up is to have a fully turbulent Living entities play a central role in bioreactors. A Hall with Poster Viewing High-Concentration flow regime in the bench scale mixing vessel. We subtle balance of operating conditions is necessary Protein Formulations compare scale-down methods for the conventional 7:00 End of Day to ensure an optimum productivity rate. Engineering stirred tank, the confined impeller stirred tank simulation can offer insight into chemical reactions, CELL & GENE THERAPY PRODUCTION (CIST), and shaker flasks. These results have THURSDAY, AUGUST 18 mixing, and multiphase flows commonly found in Cell Therapy CMC, implications both for scale-up and for many industrial bioreactors and other process equipment. ANSYS Quality & Analytics applications. Consideration of meso-mixing effects simulation tools enable designers and the operators 8:00 am Registration Opens and Morning and local concentration and turbulence effects are to boost the process line productivity. Cell Therapy Bioproduction, important considerations. Coffee Operations & Logistics 10:00 Coffee Break in the Exhibit Hall with 2:50 Establishing a Single-Use Benchtop Gene Therapy Bioproduction INCREASING PRODUCTIVITY Poster Viewing Bioreator Model to Support Flexible Manufacturing & CMC Manufacturing Strategies 8:25 Chairperson’s Remarks Strategies for Biologics Seth Kitchener, Associate Director, Upstream Stefan Schmidt, Ph.D., MBA, Vice President, Process Development, ImmunoGen, Inc. Process Science and Production, Rentschler SPONSORS & EXHIBITORS Representative bioreactor scale-down models are Biotechnology HOTEL & TRAVEL essential for successful development, transfer, and scale-up of cell culture processes. These needs REGISTRATION INFORMATION are coupled with increasing demand for speed and flexibility as programs and manufacturing BioprocessingSummit.com ◄ 14 ► COVER Upstream Processing EVENT AT A GLANCE 6th Annual PLENARY KEYNOTE SESSION SHORT COURSES Bioproduction: Scale, Bioreactors & Disposables TRAINING SEMINARS Making it Work UPSTREAM PROCESSING Optimizing Cell Culture Technology Bioproduction: Scale, Bioreactors OPTIMIZING PRODUCTON 11:45 Case Study on Microbially-Based & Disposables STRATEGIES Bioproduction Strategies Optimizing Cell Line Development Michael J. Grace, Ph.D., Head, Process & Analytical 10:45 Overcoming the Quality Challenges Development, Advaxis Immunotherapies DOWNSTREAM PROCESSING in Transferring a CHO Fed-Batch Process for 12:15 Lunch Available for Purchase in the Large-Scale Manufacturing Continuous Processing in Exhibit Hall Biopharm Manufacturing Yun Jiang, Ph.D., Principal Scientist, Project Team Leader Upstream, Drug Design & Development, Advances in Purification 1:15 Dessert Refreshment Break in the Swedish Orphan Biovitrum AB Exhibit Hall with Poster Viewing Technologies A CHO fed-batch process has been transferred Virus & Pathogen Clearance to two CMO facilities. The comparability study 1:55 End of Conference & Safety in Biologics showed that drug substance produced at the two manufacturing sites had different patterns in ANALYTICAL & QUALITY the second product main peak in the RP-HPLC Host Cell Proteins: Detection, chromatography. It was found that the difference Analysis & Removal was due to an increased level of product variants at the second site. A risk assessment of the product Early Analytical Development for variants was performed and a process change was Biotherapeutics implemented to secure a robust production process over the time. Process Characterization, Qualification & Control 11:15 Harnessing Exosome Biology to FORMULATION & STABILITY Develop the Biotherapeutics of the Future Kathryn Golden, M.Eng., Associate Director, Rapid Methods to Assess Upstream Development and Manufacturing, Codiak Quality & Stability of Biologics Biosciences Overcoming Formulation Codiak Biosciences is currently developing Challenges exosomes, natural vesicles that mediate inter- cellular communication, as both a powerful High-Concentration therapeutic modality and an advanced diagnostic Protein Formulations system. An introduction to the biology and CELL & GENE THERAPY PRODUCTION therapeutic benefits of exosome technology will be discussed. The philosophy and methodology of Cell Therapy CMC, transforming a traditionally academic production Quality & Analytics approach into an optimized full-scale manufacturing Cell Therapy Bioproduction, process will also be outlined, including updating Operations & Logistics historically nonscalable upstream and downstream methods, improving limited analytical capability, and Gene Therapy Bioproduction working with CMOs. Manufacturing & CMC Strategies for Biologics SPONSORS & EXHIBITORS HOTEL & TRAVEL REGISTRATION INFORMATION
BioprocessingSummit.com ◄ 15 ► COVER Upstream Processing EVENT AT A GLANCE 8th Annual PLENARY KEYNOTE SESSION SHORT COURSES Optimizing Cell Line Development TRAINING SEMINARS Enhancing Expression UPSTREAM PROCESSING Optimizing Cell Culture Technology THURSDAY, AUGUST 18 3:15 What is Your Cell Line Really 5:15 End of Day Bioproduction: Scale, Bioreactors Translating? Enhancing Protein Production & Disposables 11:30 am Registration Opens with Ribosomal Profiling and Systems 5:15 Registration for Dinner Short Courses Biology Analysis Optimizing Cell Line Development 12:15 pm Lunch Available for Purchase in 6:00 – 8:30 Recommended Dinner Short the Exhibit Hall Nathan E. Lewis, Ph.D., Assistant Professor, Course* DOWNSTREAM PROCESSING Department of Pediatrics, University of California, Regulatory and Scientific Base of Monoclonality Continuous Processing in San Diego 1:15 Dessert Refreshment Break in the for Generating Protein Expression Cell Banks Biopharm Manufacturing Mammalian cells channel their resources to produce Exhibit Hall with Poster Viewing * Separate registration required, see page 5 for details. Advances in Purification recombinant protein drugs, but it is unclear how INNOVATING CELL LINE recombinant genes impact global translation and if Technologies they are translated as efficiently as host genes. To FRIDAY, AUGUST 19 Virus & Pathogen Clearance DEVELOPMENT PROCESSES answer these questions, we gained a global view of 8:00 am Registration Opens and Morning & Safety in Biologics mRNA translation in IgG-producing CHO cells using 1:55 Chairperson’s Opening Remarks ribosomal profiling. We demonstrate how these data Coffee ANALYTICAL & QUALITY Nathan E. Lewis, Ph.D., Assistant Professor, can be harnessed to guide cell line development for Department of Pediatrics, University of California, protein production. CHO CELL LINE DEVELOPMENT Host Cell Proteins: Detection, San Diego Analysis & Removal 3:45 Refreshment Break 8:25 Chairperson’s Remarks Early Analytical Development for 2:00 KEYNOTE PRESENTATION: Susan Sharfstein, Ph.D., Associate Professor, Biotherapeutics Tilting at Windmills: Cell Line CELL LINE QUALITY & SCREENING Nanobioscience, Nanoscale Science and Engineering, SUNY Polytechnic Institute Process Characterization, Development Impacts on Quality, Time and Costs 4:15 A Novel Platform for High Throughput Qualification & Control Cell Line Screening & Development 8:30 Accelerated Genome Engineering of Steven Lang, Ph.D., M.B.A., Director, Early Stage CHO Cell Factories for Improved Protein FORMULATION & STABILITY Cell Culture, Genentech, Inc., a member of the Christoph Freiberg, Ph.D., Senior Scientific Consultant, Biologics, Genedata Production Rapid Methods to Assess Roche Group Co-developed with leading biopharmaceutical Helene Faustrup Kildegaard, Ph.D., Senior Quality & Stability of Biologics Advances in technology and processes have made significant improvements in cell line companies, we have implemented a dedicated cell Researcher and Co-Principal Investigator, Novo Overcoming Formulation development efficiency. However, the continued line development platform for fully automating the Nordisk Foundation Center for Biosustainability, Challenges pressure to reduce timelines and costs while clone line selection and assessment process to Technical University of Denmark delivering on quality has created imaginary increase process efficiency and quality. It supports Chinese hamster ovary (CHO) cells are widely used High-Concentration the entire cell line development workflow including in the biopharmaceutical industry as a host for the Protein Formulations giants. This talk will discuss cell line development challenges and how focusing on real issues can seeding, selection, incubation, passaging, analyzing, production of complex pharmaceutical proteins. CELL & GENE THERAPY PRODUCTION improve the value of clinical programs. and cryo-conservation and tracks the full history of Thus, accelerated genome engineering of CHO cell all clones along with product quality and analytics factories to improve product yield and quality is of Cell Therapy CMC, 2:45 Technology Toolbox for Cell Line data. It directly integrates with instruments, such as great interest. Here, our recent efforts in generating Quality & Analytics Development – Next-Generation Cell Line pipetting robots, colony pickers, and bioanalyzers. desirable CHO cell factories with predicted Cell Therapy Bioproduction, Development Technologies performance using genome engineering, systems 4:45 Quality Attributes are Critical for the biology data and high-throughput screening, will Operations & Logistics Holger Laux, Ph.D., Fellow, Novartis Lead Clone Selection and CHO Cell Culture be presented. Gene Therapy Bioproduction A novel toolbox of vector elements and novel Process Development: A Case Study engineered CHO cell lines were developed which Jianlin (Jim) Xu, Ph.D., Principal Scientist, Bristol- 9:00 Cell Line Development: results in significant increase of titer and improved Manufacturing & CMC Myers Squibb Company Barbara Potts Glycoengineering of Chinese Hamster Strategies for Biologics product quality. We have evaluated novel vector Ovary Cells for Improving Biotherapeutics’ elements with a variety of antibody projects SPONSORS & EXHIBITORS resulting in an increase of titer. Especially the Efficacies Andrew Chengyu Chung, MSE, Researcher, HOTEL & TRAVEL combination of the recently published CHO genome in combination with screening methods and cell line Chemical and Biomolecular Engineering, Johns REGISTRATION INFORMATION engineering tools has enabled the development of a Hopkins University superior CHO cell. Many biotherapeutics include post-translational BioprocessingSummit.com ◄ 16 ► COVER Upstream Processing EVENT AT A GLANCE 8th Annual PLENARY KEYNOTE SESSION SHORT COURSES Optimizing Cell Line Development TRAINING SEMINARS Enhancing Expression UPSTREAM PROCESSING Optimizing Cell Culture Technology modifications such as glycosylation. The chemical 11:00 Cell Line and Biospecimen EMERGING TECHNOLOGIES Bioproduction: Scale, Bioreactors composition of these glycans can influence drug Authentication: Challenges and Solutions effectiveness. Sialylation is one of the most for Biomedical Science 1:25 Chairperson’s Remarks & Disposables important glycan modifications due to its negative Richard M. Neve, Ph.D., Senior Research Scientist I, Christoph Freiberg, Ph.D., Senior Scientific Optimizing Cell Line Development charge and terminal location. In this study, Gilead Sciences, Inc. Consultant, Biologics, Genedata we applied genetic engineering strategies to DOWNSTREAM PROCESSING modulate the level of sialylation content on various The use of cell lines and biosamples are widespread 1:30 Greater Predictability in Establishing in biomedical research. Contamination of stocks and Continuous Processing in potential biotherapeutics. Optimal Biologic Production Systems confusion of cell line/sample origin frequently occur, Biopharm Manufacturing Using Integrated Synthetic Biology 9:30 Epigenetic Effects on Antibody resulting in wasted research funds and delaying Approaches Advances in Purification Production in CHO Cells development of important medicines for patients. This presentation outlines solutions for these issues Ian Fotheringham, Ph.D., Managing Director, Technologies Susan Sharfstein, Ph.D., Associate Professor, by defining a framework of quality controls and best Ingenza, Ltd. Nanobioscience, Nanoscale Science and Virus & Pathogen Clearance practices to prevent and detect the most common In this talk, I will describe how Ingenza develops Engineering, SUNY Polytechnic Institute & Safety in Biologics forms of cross-contamination. and applies various synthetic biology tools to enable Identifying characteristics of high productivity cell ANALYTICAL & QUALITY us to more predictably achieve efficient, soluble clones is critical for rapid cell line development. We 11:30 Authentication of Mouse, CHO, and and stable expression of active biologics using both Host Cell Proteins: Detection, evaluated epigenetic differences between parental Rat Cell Lines mammalian cell lines and microbial strains. I will clones and DHFR-amplified progeny including Analysis & Removal Jamie Almeida, Microbiologist, Biochemical Science describe scaled commercial examples that solved transcription factor activation and DNA methylation. Early Analytical Development for Division, Bioassay Methods Group, NIST customer problems and illustrate the utility of These studies suggest that transcription factor our approach. Biotherapeutics binding is altered in different families of parental cell Cell line authentication has never been more lines and progeny, providing possible strategies for necessary in the current environment. Currently, Process Characterization, there are no standards for cell line authentication 2:00 Switchable Membrane Reporter cell line engineering. Qualification & Control for nonhuman cell lines. We have developed rapid, (SwiMR) Technology for Facile Cell Line sensitive, and specific multiplex PCR assays Development FORMULATION & STABILITY 10:00 Networking Coffee Break targeting short tandem repeat markers for mouse Bo Yu, Ph.D., Co-Founder & CSO, Larix Bioscience Rapid Methods to Assess CELL LINE AUTHENTICATION and Chinese hamster cell lines, and we have Switchable Membrane Reporter (SwiMR) technology Quality & Stability of Biologics identified STR markers specific to rat. Use of these is the most efficient technology available today assays provide assurance in cell line identity for the Overcoming Formulation 10:30 Know Thy Cells: Improving for the isolation of production cell lines. It utilizes validation of downstream products. a unique switchable reporter system to facilitate Challenges Biomedical Research Reproducibility Leonard P. Freedman, Ph.D., President, Global FACS-based enrichment and isolation of high High-Concentration 12:00 pm Sponsored Presentations Biological Standards Institute producing cells. This eliminates the requirement for Protein Formulations (Opportunities Available) gene amplification and reduces cell line screening Sources of irreproducibility are often magnified time from 4-8 months to 2-3 months. The SwiMR by the explosion of high-throughput technologies, CELL & GENE THERAPY PRODUCTION 12:30 Luncheon Presentation (Sponsorship technology has been used to generate production genomics, and other data-intensive disciplines. Opportunity Available) or Lunch on Your Own CHO cells for multiple antibodies. Cell Therapy CMC, This presentation will use examples of biological Quality & Analytics materials and reagents, specifically cell lines and 1:15 Session Break Cell Therapy Bioproduction, antibodies, on the impact of irreproducibility in basic/ Operations & Logistics preclinical research and development, and how the implementation of consensus-based standards to Gene Therapy Bioproduction authenticate and certify these reagents will lead to both increased rates of reproducibility and dramatic Manufacturing & CMC returns on research funding investments. Strategies for Biologics SPONSORS & EXHIBITORS HOTEL & TRAVEL REGISTRATION INFORMATION
BioprocessingSummit.com ◄ 17 ► COVER Upstream Processing EVENT AT A GLANCE 8th Annual PLENARY KEYNOTE SESSION SHORT COURSES Optimizing Cell Line Development TRAINING SEMINARS Enhancing Expression UPSTREAM PROCESSING Optimizing Cell Culture Technology 2:30 Genetic Glycoengeneering for Bioproduction: Scale, Bioreactors Improvement of Biopharmaceuticals & Disposables Steffen Kreye, M.S., Scientist, USP Development, Glycotope GmbH Optimizing Cell Line Development Glycotope’s GEX™ platform comprises DOWNSTREAM PROCESSING a comprehensive portfolio of proprietary glycoengineered human suspension cell lines. Continuous Processing in Gene editing technologies like CRISPR/Cas9 Biopharm Manufacturing are very efficient tools to further gear up the Advances in Purification glycosylation machinery for the specific needs of Technologies human biopharmaceuticals. Besides the sialylation degree, the amount of Virus & Pathogen Clearance terminal GalNAc on the glycans greatly impacts & Safety in Biologics the pharmacokinetic parameters of glycoproteins. By using CRISPR/Cas9, we efficiently knocked out ANALYTICAL & QUALITY the respective transferases. FactorVII expressed Host Cell Proteins: Detection, in these cells showed unexpected changes in Analysis & Removal other N-glycan features leading to a recombinant FVII molecule that resembled the human plasma Early Analytical Development for derived FVII to a high extent. Biotherapeutics Process Characterization, 3:00 Close of Conference Qualification & Control FORMULATION & STABILITY Rapid Methods to Assess Quality & Stability of Biologics Overcoming Formulation Challenges High-Concentration Protein Formulations CELL & GENE THERAPY PRODUCTION Cell Therapy CMC, Quality & Analytics Cell Therapy Bioproduction, Operations & Logistics Gene Therapy Bioproduction Manufacturing & CMC Strategies for Biologics SPONSORS & EXHIBITORS HOTEL & TRAVEL REGISTRATION INFORMATION
BioprocessingSummit.com ◄ 18 ► COVER EVENT AT A GLANCE PLENARY KEYNOTE SESSION STREAM #2 SHORT COURSES TRAINING SEMINARS UPSTREAM PROCESSING Downstream Processing Optimizing Cell Culture Technology Bioproduction: Scale, Bioreactors & Disposables Process integration and process intensification are the current buzz words among biopharm scientists and executives today. Optimizing Cell Line Development This is especially prevalent and true in the downstream processing field, where economics and efficiencies are driving DOWNSTREAM PROCESSING Continuous Processing in companies to look into innovative technologies in downstream processing to meet the demands of higher upstream titers, Biopharm Manufacturing giving rise to new trends like continuous processing; advanced strategies for purification of bispecifics, vaccines and Advances in Purification Technologies recombinant proteins, and improved techniques for virus and pathogen clearance and better risk management strategies. The Virus & Pathogen Clearance weeklong Downstream Processing stream highlights exciting developments in downstream today. & Safety in Biologics ANALYTICAL & QUALITY Host Cell Proteins: Detection, Analysis & Removal Early Analytical Development for AUGUST 15-16 Biotherapeutics Process Characterization, Continuous Processing in Biopharm Manufacturing Qualification & Control FORMULATION & STABILITY Rapid Methods to Assess AUGUST 17-18 Quality & Stability of Biologics Overcoming Formulation Advances in Purification Technologies Challenges High-Concentration Protein Formulations AUGUST 18-19 CELL & GENE THERAPY PRODUCTION Cell Therapy CMC, Virus & Pathogen Clearance & Safety in Biologics Quality & Analytics Cell Therapy Bioproduction, Operations & Logistics Gene Therapy Bioproduction Manufacturing & CMC Strategies for Biologics SPONSORS & EXHIBITORS HOTEL & TRAVEL REGISTRATION INFORMATION
BioprocessingSummit.com COVER Downstream Processing EVENT AT A GLANCE 2nd Annual PLENARY KEYNOTE SESSION SHORT COURSES Continuous Processing in Biopharm Manufacturing TRAINING SEMINARS UPSTREAM PROCESSING Reinventing Process Architecture with Integration, Intensification and Automation Optimizing Cell Culture Technology Bioproduction: Scale, Bioreactors MONDAY, AUGUST 15 1:45 Medium Development for Next- 3:45 Selected Poster Presentation: & Disposables Generation Perfusion Processes Evaluation of Flow-VPE for In-Line 8:00 am Short Course Registration Jochen B. Sieck, Ph.D., Lab Head, Cell Culture Concentration Measurement of Monoclonal Optimizing Cell Line Development Media R&D, Process Solutions R&D, Merck KGaA Antibodies During Chromatography 9:00 – 11:30 Recommended Morning DOWNSTREAM PROCESSING Perfusion processes have fundamentally different Pranali Shah, MSc., Senior Associate, Purification Short Course* Continuous Processing in requirements for cell culture media, compared Process Development, Amgen Inc. Advanced Process Control Strategies to fed-batch processes. Desirable characteristics Biopharm Manufacturing in Bioprocessing also depend on the type of perfusion process 4:00 A Membrane-Less Cell Retention Advances in Purification * Separate registration required, see page 5 for details. that is targeted. In this talk we will present how Device Based on Inertial Sorting for Technologies different manifestations of perfusion impact these Perfusion Cultures 11:30 Main Conference Registration Opens requirements and present data on how they can be Virus & Pathogen Clearance Taehong Kwon, MSc, Research Assistant, Electrical addressed in medium development. Engineering and Computer Science, MIT & Safety in Biologics CONTINUOUS UPSTREAM 2:15 Scalability of Growth Characteristics Membrane-based cell retention technologies PROCESSING (e.g., hollow fiber membranes) are currently used ANALYTICAL & QUALITY and Product Quality: Efficient Downscale Host Cell Proteins: Detection, in perfusion cultures despite the limitations of 1:00 pm Chairperson’s Opening Remarks Perfusion Bioprocess Development Using membrane fouling and clogging. We developed a Analysis & Removal Andrew Sinclair, MSc., FREng, President and DOE Studies membrane-less clog-free retention device based Early Analytical Development for Founder, Biopharm Services Steffen Kreye, MSc, Scientist, USP Development, on inertial sorting, and used it for IgG-producing Biotherapeutics Glycotope GmbH perfusion CHO cultures. This talk will introduce 1:10 KEYNOTE PRESENTATION: The sedimentation based down-scale perfusion the novel cell retention device and discuss its Process Characterization, Upstream and Downstream Approaches system SAM (10mL reactor volume) has been challenges and promises for continuous processing Qualification & Control to Continuous Biopharmaceutical developed to characterize the upstream process in biopharmaceutical manufacturing. Manufacturing: A Journey Not a parameters and their influence on product quality. FORMULATION & STABILITY 4:30 Breakout Discussions Destination Using DoE studies we gain a highly efficient Rapid Methods to Assess method for media development as well as process Quality & Stability of Biologics John Knighton, MBA, Head, API Large Molecule, optimization to achieve higher cell densities and 5:30 Grand Opening Reception in the Pharmaceutical Development and Manufacturing Exhibit Hall with Poster Viewing Overcoming Formulation higher productivities. Scalability and reproducibility Sciences, Janssen R&D of perfusions bioreactors (10mL – 1000L) will be Challenges 7:00 End of Day The topic will explore broadly the highlighted with data of the fully human, high High-Concentration biopharmaceutical industry’s different yield production and glycooptimization platform Protein Formulations viewpoints of continuous manufacturing, GlycoExpress (GEX). TUESDAY, AUGUST 16 specifically providing some Janssen continuous CELL & GENE THERAPY PRODUCTION manufacturing examples. For some, it is 2:45 Refreshment Break 7:30 am Registration Opens and Morning Cell Therapy CMC, more holistic view from vial thaw to filled drug Coffee product in one facility. For others like Janssen, 3:15 Perfusion Process Applicability Quality & Analytics continuous manufacturing is comprised Jacob Jensen, Senior Manager, Manufacturing CONTINUOUS DOWNSTREAM Cell Therapy Bioproduction, of continuous aspects of upstream and Sciences, Biogen Operations & Logistics downstream as the technology develops and as Perfusion processes extends the time in the PROCESSING the need for more plant efficiency increases. manufacturing bioreactor and may not be directly Gene Therapy Bioproduction 7:55 Chairperson’s Remarks applicable to conventional fed-batch processing. Robert Dream, PE, CPIP, Managing Director, HDR Manufacturing & CMC This talk will focus on how the benefits of perfusion Company, LLC Strategies for Biologics can be integrated in a fed-batch process to increase product output. SPONSORS & EXHIBITORS HOTEL & TRAVEL REGISTRATION INFORMATION
BioprocessingSummit.com ◄ 20 ► COVER Downstream Processing EVENT AT A GLANCE 2nd Annual PLENARY KEYNOTE SESSION SHORT COURSES Continuous Processing in Biopharm Manufacturing TRAINING SEMINARS UPSTREAM PROCESSING Reinventing Process Architecture with Integration, Intensification and Automation Optimizing Cell Culture Technology 9:30 Comparison of Batch and Continuous challenges and we also identified strategies on how 8:00 KEYNOTE PRESENTATION: Bioproduction: Scale, Bioreactors Multi-Column Protein A Capture Processes to further advance this concept. Continuous Precipitation-Based & Disposables Thomas Mueller-Spaeth, Ph.D., Professor, Institute Processes for Protein Purification 11:00 PAT Strategies in Support of Process for Chemical and Bioengineering, ETH Zurich Optimizing Cell Line Development Todd Przybycien, Ph.D., Professor, Chemical Monitoring and Control to Enable Rapid Protein A affinity chromatography is used as a Engineering and Biomedical Engineering, DOWNSTREAM PROCESSING powerful purification platform in industry for capture Product Release Carnegie Mellon University Continuous Processing in of a number of therapeutic proteins including mAbs Jeffrey Doyle, Manager, PAT Projects, Pfizer, Inc. and fusion proteins from cell culture harvest. In the Continuous bioprocessing offers potential to Biopharm Manufacturing 8:30 Continuous Manufacturing, Hybrid last years, the stationary phase capacity utilization enhance productivity and product quality uniformity Advances in Purification Options Transitioning from Batch to and productivity of affinity chromatography has been while simultaneously decreasing facility footprint Technologies Continuous: Economics and Operational significantly improved by newly introduced multi- and associated operational overhead. Advances in Implications technology and increasing commercial pressures Virus & Pathogen Clearance column sequential loading processes for continuous Andrew Sinclair, MSc., FREng, President and mAb purification. This presentation provides a side- are leading to an increased interest in continuous & Safety in Biologics Founder, Biopharm Services Ltd. by-side comparison Protein A capture processes processing across the biopharmaceutical sector. ANALYTICAL & QUALITY This presentation will assess the reality of with 1 to 4 columns based on a chromatographic The advancement of continuous bioprocessing transitioning to the hybrid option by providing simulation model that was calibrated and validated presents a range of opportunity and challenges, Host Cell Proteins: Detection, insights on where this makes practical and using experimental capture data. The processes including the use of Process Analytical Technology Analysis & Removal economic sense within a process. This allows us were compared in terms of resin costs, productivity, (PAT) for process characterization, process control, Early Analytical Development for to identify those factors that require optimization/ buffer consumption and equipment complexity. The and process robustness, in support of a Rapid Product Release (RPR) strategy. Biotherapeutics development in terms of the potential now and study shows significant advantages of the twin- for the future. Here the focus is on describing the column process over batch chromatography and Process Characterization, approaches to hybrid downstream processing in other multi-column processes. 11:30 Validation, Continuous Verification Qualification & Control terms of the mode of operation, integrating batch and Compliance of Continuous processing into continuous operations and looking 10:00 Coffee Break in the Exhibit Hall with Manufacturing Operations FORMULATION & STABILITY at the impact on buffer supply to the process. Poster Viewing Michelle Bailey, Ph.D., Head, Validation of Rapid Methods to Assess Continuous Manufacturing and Process Automation, Quality & Stability of Biologics 9:00 Continuous Chromatography for MONITORING, CONTROL & Vertex Pharmaceuticals Purification of Virus and Virus-Like Particles Overcoming Formulation VALIDATION OF CONTINUOUS The pharmaceutical industry is adopting continuous Targeting Vaccine and Gene Therapy Challenges PROCESSES manufacturing models to drive efficiency and Applications higher quality. These new technologies require High-Concentration Ricardo Silva, Ph.D., Researcher, Downstream 10:30 Implementation of Model Predictive effective approaches for validation. This presentation Protein Formulations Processing Lab, Animal Cell Technology Unit, iBET – Fermentation Process Control Strategies - discusses challenges encountered and approaches Instituto de Biologia Experimental e Tecnologica taken to validate continuous manufacturing for CELL & GENE THERAPY PRODUCTION Experienced Challenges and Strategies on multiproduct solid dosage product and preparing for Novel biopharmaceutical products are a How to Solve These Problems Cell Therapy CMC, challenging task for downstream processing. a successful regulatory inspection. Gerald Striedner, Ph.D., Assistant Professor, Quality & Analytics Alternative purification strategies that can Sponsored By Biotechnology, University of Natural Resources and 12:00 pm A High improve the purification yield, such as continuous Cell Therapy Bioproduction, Life Sciences Performance Integrated Operations & Logistics chromatography, are regarded nowadays as enabling technologies to overcome the capacity bottleneck We established data driven models for real time and Disposable Clarification Gene Therapy Bioproduction in biomanufacturing. The current talk will focus on prediction of relevant process variables as basis Solution for Continuous Bioprocessing for an advanced process control strategy. We used the development of multi-column chromatographic Mike Collins, Principal Research Engineer, Pall Life the predicted biomass in the process to calculate Manufacturing & CMC systems aimed at the purification of gene therapy Sciences the inducer feed profile that allowed for setting Strategies for Biologics vectors and enveloped virus-like particles produced There is a growing interest within the a constant inducer to biomass ratio and thereby using insect cell-based expression system for biopharmaceutical industry in adopting continuous SPONSORS & EXHIBITORS indirect control of the product formation rate. During vaccine applications. processing in place of current batch processing implementation and evaluation of this advanced HOTEL & TRAVEL for biological molecules and primarily monoclonal process control strategy we experienced various REGISTRATION INFORMATION
BioprocessingSummit.com ◄ 21 ► COVER Downstream Processing EVENT AT A GLANCE 2nd Annual PLENARY KEYNOTE SESSION SHORT COURSES Continuous Processing in Biopharm Manufacturing TRAINING SEMINARS UPSTREAM PROCESSING Reinventing Process Architecture with Integration, Intensification and Automation Optimizing Cell Culture Technology Bioproduction: Scale, Bioreactors antibodies (mAbs). Expectations from this transition Sciences Technology & Innovation, Biopharma Global 4:45 Closing Presentation are multiple including reduced product cost of Manufacturing & Supply, Merck Serono & Disposables Integrated Continuous Biomanufacturing goods, capital expenses and facility foot print while The objective of this presentation will be to provide Enables More Efficient Cost-Effective Drug Optimizing Cell Line Development process productivity and flexibility is increased. But invaluable insight into the design and development Manufacturing and Improved Process the adoption of continuous bioprocessing requires of a fully integrated platform manufacturing Safety DOWNSTREAM PROCESSING first to achieve a smooth transition for each process process operated in continuous mode. Challenges Continuous Processing in step from batch to continuous. The clarification is of integrating discrete unit operations to create a Robert Dream, PE, CPIP, Managing Director, HDR Biopharm Manufacturing a key early step within the process which poses compact continuous processing drug substance Company, LLC the challenge of reducing the cost and foot print manufacturing solution will be reviewed. Process The importance and value of continuous Advances in Purification of filtration in the context of a continuous mAb design considerations and enabling technologies bioprocessing has economic and sustainability Technologies purification process. required to monitor, control and optimize the advantages and due to the modular nature of Virus & Pathogen Clearance process will be discussed. continuous bioprocesses it means that the 12:30 Luncheon Presentation (Sponsorship industry is able to adapt more rapidly to changing & Safety in Biologics Opportunity Available) or Lunch on Your Own 3:00 Integration of Single-Use Technologies market demands. Continuous manufacturing is as ANALYTICAL & QUALITY and Continuous Processing for the productive and with a much smaller footprint of the 1:15 Dessert Refreshment Break in the Manufacturing of Therapeutic Proteins manufacturing plant, avoiding multiple non-value Host Cell Proteins: Detection, Exhibit Hall with Poster Viewing added unit operations. In essence, the investment Jorgen Magnus, Ph.D., Project Manager, R&D, Bayer Analysis & Removal for a continuous plant is much smaller compared to Technology Services INTEGRATED UP- AND DOWN-STREAM a batch-operated one. Early Analytical Development for Bayer is developing a novel production concept Biotherapeutics CONTINUOUS BIOMANUFACTURING for monoclonal antibodies within the Mobidik 5:15 End of Conference project. All surfaces in contact with the product Process Characterization, 1:55 Chairperson’s Remarks Qualification & Control are in single-use technology and the downstream 5:15 Registration for Dinner Short Course Todd Przybycien, Ph.D., Professor, Chemical processing is run as a truly continuous process FORMULATION & STABILITY Engineering and Biomedical Engineering, Carnegie without intermediate storage. A process control 6:00 – 8:30 Recommended Dinner Short Mellon University Rapid Methods to Assess system and on-line / at-line PAT are developed to Course* give a high level of automation. GMP related issues Quality & Stability of Biologics 2:00 Manufacturing Sponsored By Purification of Advanced Medicine are addressed at an early stage. Therapy Products Overcoming Formulation Technologies to Enable * Separate registration required, see page 5 for details. Challenges Process Intensification 3:30 Refreshment Break in the Exhibit Hall Jim Neville, Director, Technology Management- with Poster Viewing High-Concentration Americas’, MilliporeSigma Protein Formulations The biopharmaceutical industry is adopting a 4:15 Challenges in the Development of CELL & GENE THERAPY PRODUCTION more strategic view toward manufacturing, Continuous Processes for Vaccines seeking solutions that offer increased productivity Daniel C. Vellom, Ph.D., Senior Director, Global Cell Therapy CMC, and improved economics without sacrificing Technology Innovation, Sanofi Pasteur Biologics Quality & Analytics process robustness. The industry addresses Vaccine developers pursuing continuous Cell Therapy Bioproduction, these challenges through process intensification manufacturing need to consider the nature Operations & Logistics efforts including unit operation optimization, linked of the batch production process and the and continuous processing. This presentation economics supporting the transition to a new Gene Therapy Bioproduction provides insight into upstream and downstream mode of manufacturing. Will the “new” product intensification approaches to improve processes. be comparable to the existing vaccine, or be Manufacturing & CMC Examples include high producing and stable considered a next-generation product in the eyes Strategies for Biologics cell lines, column size reduction, product purity of the regulatory agencies? This presentation will SPONSORS & EXHIBITORS improvement and enabling a continuous process. outline these challenges and propose a general approach to development of a continuous vaccine HOTEL & TRAVEL 2:30 Building an Integrated Continuous production process, and new tools that can enable REGISTRATION INFORMATION Manufacturing Platform such development. Jonathan Souquet, Ph.D., Head, Biotech Process BioprocessingSummit.com ◄ 22 ► COVER Downstream Processing EVENT AT A GLANCE 3rd Annual PLENARY KEYNOTE SESSION SHORT COURSES Advances in Purification Technologies TRAINING SEMINARS Economics and Efficiencies for Next-Generation Biomanufacturing UPSTREAM PROCESSING TUESDAY, AUGUST 16 DEFINING DESIGN SPACE Optimizing Cell Culture Technology KEYNOTE SESSION Bioproduction: Scale, Bioreactors 6:00 – 8:30 Recommended Dinner Short 9:30 Defining Process Design Space for a & Disposables Course* 8:15 From Purification to Vaccine Multimodal Chromatography Purification Step by Custom Design Optimizing Cell Line Development Purification of Advanced Medicine Development: A Challenging but Therapy Products Rewarding Journey Jie Chen, Ph.D., Senior Scientist, Process Development, Bristol-Myers Squibb DOWNSTREAM PROCESSING * Separate registration required, see page 5 for details. Yan-Ping Yang, Ph.D., Head, Bioprocess R&D Continuous Processing in North America, Sanofi Pasteur A process characterization study was performed to gain understanding of the multimodal Biopharm Manufacturing WEDNESDAY, AUGUST 17 The vaccine industry has high risk, long cycle chromatography (MMC) purification process and time, and requires approximately 12 years Advances in Purification to map the design space. Seven factors out of 7:00 am Registration Opens and Morning bringing a product from pre-clinical to licensure thirty-eight process parameters were identified to Technologies Coffee at a cost of ~$1 billion. Purification of vaccine be important. Custom design was employed to Virus & Pathogen Clearance candidates to achieve consistent product purity study multi-dimensional combination of operational 8:05 Chairperson’s Opening Remarks and quality in a timely manner is an integral & Safety in Biologics variables for mapping of the process design Christoph Brandenbusch, Ph.D., Group Leader, part of vaccine product development process. space. A robust operating window was identified ANALYTICAL & QUALITY Department of Biochemical and Chemical This presentation focuses on the applications of for this multimodal chromatography process that Engineering, Laboratory of Thermodynamics, TU cutting edge purification technologies to facilitate Host Cell Proteins: Detection, enabled sufficient impurity removal while ensuring Dortmund University vaccine process development and move faster to Analysis & Removal acceptable process yield. clinical evaluations. Early Analytical Development for 10:00 Coffee Break in the Exhibit Hall with 9:00 Positioning Downstream Processing Biotherapeutics Poster Viewing to Successfully Conquer Future Challenges Process Characterization, Qualification & Control John Pieracci, Ph.D., Director, Purification CONTINUOUS AQUEOUS TWO-PHASE Development, Biogen FORMULATION & STABILITY The biotechnology industry will be facing EXTRACTION considerable challenges in the future to provide Rapid Methods to Assess 10:45 Continuous Aqueous Two-Phase wider access to treatments or cures to diseases Quality & Stability of Biologics affecting millions of people. The solution to Extraction of Proteins Part I – Estimating Overcoming Formulation supplying these large patient populations will Process Windows and Industrial Challenges come from new classes of therapeutic drugs Applicability possessing greater specificity, higher potency Christoph Brandenbusch, Ph.D., Group Leader, High-Concentration or better manufacturability and by driving up the Department of Biochemical and Chemical Protein Formulations productivity of the manufacturing process for Engineering, Laboratory of Thermodynamics, TU CELL & GENE THERAPY PRODUCTION existing drugs. This presentation will review the Dortmund University impact that these potential options could have Increasing product titers in pharmaceutical protein Cell Therapy CMC, on the development of downstream processes. production lead to a demand for novel workup Quality & Analytics Substantial pressure will be on downstream strategies (eg: protein extraction by Aqueous Cell Therapy Bioproduction, scientists to support more productive batch 2-Phase System). However the effort and costs Operations & Logistics processes in order to leverage the full potential required in selecting appropriate ATPS and the of existing manufacturing infrastructure, as well determination of process windows hinders the Gene Therapy Bioproduction as develop continuous downstream processes industrial applicability. To enable estimations on the for alternative manufacturing plant designs. New applicability of this workup strategy, hybrid ShortCut Manufacturing & CMC downstream process platforms will be needed models to calculate partition coefficients have to Strategies for Biologics to enable new classes of drugs or to support be available. SPONSORS & EXHIBITORS the application of more productive expression systems to current modalities. Identifying HOTEL & TRAVEL existing downstream technologies that can be readily implemented or investing in breakthrough REGISTRATION INFORMATION technologies that could take time to mature, but could represent significant leaps, will be key. BioprocessingSummit.com ◄ 23 ► COVER EVENT AT A GLANCE Downstream Processing PLENARY KEYNOTE SESSION 3rd Annual SHORT COURSES TRAINING SEMINARS Advances in Purification Technologies UPSTREAM PROCESSING Economics and Efficiencies for Next-Generation Biomanufacturing Optimizing Cell Culture Technology Bioproduction: Scale, Bioreactors 11:15 Continuous Aqueous Two-Phase 1:45 Chairperson’s Remarks Instituto de Biologia Experimental e Tecnologica & Disposables Extraction of Proteins Part II – Separation Yan-Ping Yang, Ph.D., Head, Bioprocess R&D North The use of human mesenchymal stem cells (hMSC) Optimizing Cell Line Development Principle and Novel Techniques America, Sanofi Pasteur in autologous and allogeneic therapies has been Gerhard Schembecker, Ph.D., Professor, Biochemical increasing over the last decade. To be applied in a 1:50 Creating an Efficient DOWNSTREAM PROCESSING and Chemical Engineering, TU Dortmund University clinical setting, hMSC need to comply with specific Biopharmaceutical Factory: Protein requirements in terms of identity, potency and Continuous Processing in Aqueous Two Phase Extraction (ATPE) offers purity. This talk will focus on the improvement of Biopharm Manufacturing a gentle and biocompatible environment to Purification Using a Self-Cleaving Split established tangential flow filtration-based washing separate industrially interesting enzymes from Intein Advances in Purification strategies, by increasing hMSC purity, using complex mixtures. The presentation will introduce Merideth Cooper, MSc, Chemical and Biomolecular negative mode expanded bed adsorption (EBA) Technologies the separation principle and existing and novel Engineering, Ohio State University chromatography with a new multimodal prototype Virus & Pathogen Clearance techniques will be discussed particularly in the light Although affinity tags are commonly used in matrix based on core-shell bead technology. & Safety in Biologics of continuous operation. Besides single and multi- laboratory settings, tagged proteins cannot be stage mixer settler setups innovative processes used as therapeutics because of the potential 3:20 Accelerating Bispecific Drug Discovery ANALYTICAL & QUALITY immobilizing one of the two aqueous phases either immunogenicity of the tag. In this work, we Using Enhanced Purification and Analytical Host Cell Proteins: Detection, in the pores of particles or by use of a centrifugal describe a novel self-cleaving tag technology, based force fields will be investigated. on a highly modified split-intein cleaving element. Techniques Analysis & Removal Daniel Yoo, Scientist, Biologics, Amgen Sponsored By This approach is convenient and effective for the Early Analytical Development for 11:45 Amsphere A3: purification of traceless target glycoproteins from Isolating high quality proteins from other undesired Biotherapeutics Considerations of Bead mammalian cell culture, and is currently being byproducts of expression, including aggregates, half antibodies, homodimer molecules, and mispaired Process Characterization, and Pore structure, Surface applied in a BioMOD platform device for on-demand biologics production. species, poses significant purification challenges Qualification & Control Chemistries, and Ligand Design for Affinity Resins due to the similarities in physical properties of these 2:20 The PA Tag Toolbox Offers a System for molecules. Here, I present strategies we have FORMULATION & STABILITY Masayoshi Nagaya, Business Strategy Manager, Easy Affinity Isolation, Robust Detection, explored to accelerate bispecific drug discovery Rapid Methods to Assess Bioprocess, JSR Life Sciences and Recyclable Immobilization of High- using a combination of higher throughput screening, Quality & Stability of Biologics Industry needs, patent expirations of ligand Value Target Proteins automated sample handling, and enhanced design and market dynamics have accelerated the purification and analytical techniques to identify Overcoming Formulation Junichi Takagi, Ph.D., Professor, Institute for Protein development and availability of many protein affinity higher quality therapeutic candidates. Challenges resins. Technology providers must consider a range Research, Osaka University High-Concentration of design parameters such as resin backbone, bead We recently developed a novel and versatile epitope 3:50 Refreshment Break in the Exhibit Hall Protein Formulations size, pore size, surface chemistry, ligand construct tag system dubbed “PA tag”. It is a dodecapeptide with Poster Viewing and how they relate to performance criteria such tag recognized by an antibody NZ-1, with a CELL & GENE THERAPY PRODUCTION as DBC, life time, caustic stability, and column characteristic slow dissociation kinetics. PA tag 4:45 Plenary Keynote Session (see page 3 Cell Therapy CMC, packing among others. The balance between resin enables purification, detection, and stable and for details) reversible capturing of high value target proteins Quality & Analytics design and performance will be illustrated through a case study. produced in mammalian cells. We also find that the 6:00 Networking Reception in the Exhibit Cell Therapy Bioproduction, PA tag can be “inserted” into a protein domain, Hall with Poster Viewing Operations & Logistics 12:15 Luncheon Presentation (Sponsorship which adds unique advantage over the existing tagging systems. 7:00 End of Day Gene Therapy Bioproduction Opportunity Available) or Lunch on Your Own 1:00 Session Break 2:50 Application of Negative Mode Manufacturing & CMC Expanded Bed Chromatography to Improve Strategies for Biologics IMPROVEMENTS IN Filtration-Based Washing Strategies of SPONSORS & EXHIBITORS CHROMATOGRAPHY & ANALYTICAL Human Mesenchymal Stem Cells HOTEL & TRAVEL TECHNIQUES FOR PURIFICATION Barbara Cunha, MSc, Animal Cell Technology, iBET – REGISTRATION INFORMATION
BioprocessingSummit.com ◄ 24 ► COVER 3rd Annual EVENT AT A GLANCE Downstream Processing PLENARY KEYNOTE SESSION Advances in Purification Technologies SHORT COURSES Economics and Efficiencies for Next-Generation Biomanufacturing TRAINING SEMINARS UPSTREAM PROCESSING THURSDAY, AUGUST 18 9:15 Poster Highlight II: To Be Announced 11:45 Navigating the Downstream Processing Challenges for Novel Optimizing Cell Culture Technology Sponsored By 8:00 am Registration Opens and Morning 9:30 Inline Protein Monoclonal and Bispecific Antibodies Bioproduction: Scale, Bioreactors Concentration for Coffee Chen Wang, Ph.D., Principal Scientist, Process & Disposables Downstream Processing Sciences, Abbvie Bioresearch Center Optimizing Cell Line Development PURIFICATION OF EMERGING Ramsey Shanbaky, Product Manager, Sales, C As we expand our biotherapeutic product portfolio, BIOMOLECULES Technologies, Inc significant processing limitations have arisen DOWNSTREAM PROCESSING Accurate concentration measurement is critical with the existing platform for some of the novel Continuous Processing in 8:25 Chairperson’s Remarks during downstream processes. Providing real-time monoclonal and bi-specific antibodies such as dual Biopharm Manufacturing Guy de Roo, Ph.D., Project Leader, Downstream concentration during protein purification processes variable domain immunoglobulins (DVD-IgsTM). expedites process development and reduces risk Through exploring unconventional chromatographic Processing, Synthon BV Advances in Purification in manufacturing processes. The FlowVPE is a methods, adopting state-of-the-art purification Technologies 8:30 KEYNOTE PRESENTAITON: new tool to provide real-time concentration during technologies and understanding protein-protein Virus & Pathogen Clearance downstream processes. This talk will discuss interaction mechanism, we were able to develop Development and Manufacturing of applications in chromatography and UF/DF. & Safety in Biologics MCLA-128 and MCLA-117 Biclonics® effective strategy and toolbox to overcome 10:00 Coffee Break in the Exhibit Hall with processing challenges for molecules with platform Common Light Chain Bispecific ANALYTICAL & QUALITY Poster Viewing fit concern. Antibodies Host Cell Proteins: Detection, Lex Bakker, Ph.D., Senior Vice President & CDO, 10:45 Process Development of the 12:15 Lunch Available for Purchase in the Analysis & Removal Merus Biopharmaceuticals Antibody-Drug Conjugate (ADC) SYD985 - A Exhibit Hall Early Analytical Development for MCLA-128 is an ADCC-enhanced bispecific IgG1 Case Study Biotherapeutics antibody targeting HER2 and HER3. MCLA-117 Guy de Roo, Ph.D., Project Leader, Downstream 1:15 Dessert Refreshment Break in the is a T cell engaging bispecific antibody targeting Processing, Synthon BV Exhibit Hall with Poster Viewing Process Characterization, CD3 and CLEC12A, a novel AML-associated SYD985 is an antibody-drug conjugate (ADC) based Qualification & Control antigen. MCLA-128 and MCLA-117 are produced 1:55 End of Conference on trastuzumab and a cleavable linker-duocarmycin using Merus’ proprietary CH3 heterodimerization FORMULATION & STABILITY payload. A case study will be presented in which technology. Both Biclonics® product leads are a hydrophobic interaction chromatography (HIC) Rapid Methods to Assess manufactured using IgG1 platform approaches. Quality & Stability of Biologics purification process was developed allowing removal of the undesired antibody species together with Overcoming Formulation 9:00 Poster Highlight I: A Split Intein unbound linker-drug. It was possible to elute the Challenges Based Platform for the Purification of ANY product (SYD985) using mild conditions without requirement for any organic solvent. The HIC High-Concentration Biological Therapeutic Protein purification step was scaled up, demonstrating Protein Formulations Ashwin Lahiry, Ph.D. Candidate, Wood Lab, The Ohio State University consistency and robustness. CELL & GENE THERAPY PRODUCTION Currently, there is no simple, low cost platform 11:15 Preparative Separation of VLP and for the purification of non-antibody therapeutic Cell Therapy CMC, Extra Cellular Particles (Exosomes) Quality & Analytics proteins. Our lab has developed an affinity tag-based platform for purification of ANY recombinant protein. Alois Jungbauer, Ph.D., Professor, Laboratory of Cell Therapy Bioproduction, This platform utilizes an engineered self-cleaving Protein Technology and Downstream Processing, Operations & Logistics split-intein tag that enables tagless and traceless Austrian Center of Biotechnology, Department of Biotechnology, University of Natural Resources and Gene Therapy Bioproduction purification of recombinant proteins. We have successfully demonstrated the utility of this split- Life Sciences Manufacturing & CMC intein platform in E. coli, Mammalian and CHO IVT Enveloped VLPs are interesting candidates for expression systems. vaccines and cancer immunotherapy. They have Strategies for Biologics a size of about 100 -200 nm which is similar to SPONSORS & EXHIBITORS exosomes and other extra cellular particles. The surface properties are very similar because VLPs HOTEL & TRAVEL and extra cellular particles are budded from the host REGISTRATION INFORMATION cell. A strategy is shown who solve this extremely challenging purification task using HIV-gag VLP expressed in CHO cells as model. BioprocessingSummit.com ◄ 25 ► COVER Downstream Processing EVENT AT A GLANCE 2nd Annual PLENARY KEYNOTE SESSION SHORT COURSES Virus and Pathogen Clearance and Safety in Biologics TRAINING SEMINARS Adventitious Agent Contamination, Risk Mitigation, New Technologies and Case Studies UPSTREAM PROCESSING Optimizing Cell Culture Technology Bioproduction: Scale, Bioreactors THURSDAY, AUGUST 18 The purpose of this talk is to provide an overview anion exchange & Disposables of key features of this new publication and make • Single-use pathogen removal 11:30 am Registration Opens the audience aware of the value it brings to those Optimizing Cell Line Development practicing the art of viral clearance. 5:15 End of Day 12:15 pm Lunch Available for Purchase in DOWNSTREAM PROCESSING the Exhibit Hall 3:15 Protein A: The Untold Story 5:15 Registration for Dinner Short Course Continuous Processing in Pete Gagnon. Vice President, Process Sciences, Biopharm Manufacturing 1:15 Dessert Refreshment Break in the Avid Bioservices, Inc. FRIDAY, AUGUST 19 Exhibit Hall with Poster Viewing Advances in Purification This presentation will review recent discoveries about the way protein A really works: how it Technologies 8:00 am Registration Opens and Morning VIRAL SAFETY, RISK ASSESSMENT & affects IgG, why it is generally unable to reduce Coffee Virus & Pathogen Clearance MANAGEMENT host contaminants below 500 ppm, and how to & Safety in Biologics enable it to reduce them below 10 ppm. New data VIRAL SAFETY: CHARACTERIZATION & 1:55 Chairperson’s Opening Remarks will be shown from case studies conducted with ANALYTICAL & QUALITY Mark Plavsic, Ph.D., Senior Director, Product prospective biosimilar Humira, Herceptin, and other INACTIVATION antibodies. Examples will demonstrate consistently Host Cell Proteins: Detection, Biosafety, Industrial Operations, Sanofi 8:25 Chairperson’s Remarks Analysis & Removal better purification with 2 chromatography steps than Donald Jarvis, Ph.D., Professor, Molecular Biology, 2:00 Considerations for Use of New can be achieved by the traditional 3-step platform. Early Analytical Development for University of Wyoming & President, GlycoBac LLC Technologies for Adventitious Virus Biotherapeutics 3:45 Genetic Engineering of Sponsored By Detection CHO Cells for Viral Resistance 8:30 Redevelopment of a Viral Filtration Process Characterization, Arifa Khan, Ph.D., Supvy Microbiologist, Viral to Minute Virus of Mice (MVM) Step for a Large and Complex Monoclonal Qualification & Control Products, FDA CBER Joaquina Mascarenhas, Ph.D., Senior Research & Antibody Based Fusion Protein FORMULATION & STABILITY Several advanced nucleic acid based technologies Development Scientist, MilliporeSigma George Enriquez, Ph.D., Senior Process have emerged with capabilities of broad virus We have engineered a host cell line resistant Development Specialist, Bio Process Department, Rapid Methods to Assess detection. High throughput sequencing (HTS) to MVM infection, while maintaining desired Shire Quality & Stability of Biologics has the potential for identification of both known productivity and product quality profiles. This A quality by design (QbD) based, late stage and novel viruses without prior knowledge of Overcoming Formulation approach can be applied to different CHO host purification process development approach was virus genome sequences. Therefore, HTS may Challenges cell lines as well as therapeutic protein producing used for a 300kD, complex monoclonal antibody complement the currently recommended testing recombinant cell lines. This is another level of (Mab) based fusion protein. A manufacturability High-Concentration for the presence of unknown viruses, including “defense” to current viral risk mitigation strategies. risk assessment identified viral filter fouling as a Protein Formulations latent and endogenous viruses that may not high risk to a robust, cost effective manufacturing be detected by the conventional assays. This CELL & GENE THERAPY PRODUCTION 4:00 Refreshment Break operation. Through systematic filter scouting and presentation will describe the need for new virus optimization development work, the combination of Cell Therapy CMC, detection methods, challenges of using HTS, and 4:15 Improvements and Selection of a pre-filter and a viral filter was found to deliver high Quality & Analytics ongoing efforts toward filling the critical gaps Downstream Viral Clearance Technology throughput, high recovery & short processing time. for using new virus detection technologies for Cell Therapy Bioproduction, Pete Gagnon, Vice President, Process Sciences, detection of adventitious viruses. 9:00 Characterization of an Sf-rhabdovirus Operations & Logistics Avid Bioservices Inc. Negative Insect Cell Line 2:45 Design, Evaluation, and This panel will discuss technology advancements Gene Therapy Bioproduction available to biological drug manufacturers to meet Donald Jarvis, Ph.D., Professor, Molecular Biology, Characterization of Viral Clearance viral clearance standards. Protein react to these University of Wyoming & President, GlycoBac LLC Manufacturing & CMC Procedures: USP Chapter <1050.1> methods will be explored and discussed. The recent discovery that insect cell lines derived Strategies for Biologics Mark Plavsic, Ph.D., Head of Process Development • Nanofiltration technology advancements resulting from Spodoptera frugiperda (Sf) are contaminated SPONSORS & EXHIBITORS and Manufacturing, Torque Therapeutics in the removal of large virus/pathogens with a rhabdovirus raised questions about their The new USP Chapter <1050.1> on viral clearance biosafety as a commercial recombinant protein • Inactivation and removal- chromatography / HOTEL & TRAVEL will be published in 2016. It is meant to supplement production substrate. To address this problem, we REGISTRATION INFORMATION USP Chapter <1050> and the ICH guidance Q5A. created Sf-rhabdovirus-negative (Sf-RVN) cells,
BioprocessingSummit.com ◄ 26 ► COVER EVENT AT A GLANCE Downstream Processing PLENARY KEYNOTE SESSION 2nd Annual SHORT COURSES TRAINING SEMINARS Virus and Pathogen Clearance and Safety in Biologics UPSTREAM PROCESSING Adventitious Agent Contamination, Risk Mitigation, New Technologies and Case Studies Optimizing Cell Culture Technology
Bioproduction: Scale, Bioreactors which have no detectable trace of this adventitious of acute gastroenteritis in the U.S., estimated to Nanotechnology, Wallenberg Academy Fellow, & Disposables agent. In this presentation, we will describe the afflict 21 million people per year. For manufacturing Nanotechnology and Functional Materials, Optimizing Cell Line Development features of Sf-RVN cells as an alternative host for of a vaccine candidate, the baculovirus expression Department of Engineering Sciences, Uppsala use in the baculovirus-insect cell system. system has been used for production of two distinct University DOWNSTREAM PROCESSING norovirus virus-like particles (VLPs). Processing of 2:00 Raw Materials as the Source of Porcine Continuous Processing in 9:30 Downstream Process Development VLPs employs enveloped virus inactivation and Studies orthogonal chromatography steps. Viral clearance Circovirus, Porcine Hepatitis E Virus and Biopharm Manufacturing David Cetlin, Founder & CEO, MockV Solutions LLC validation has been conducted for select unit Mycoplasmas, Strategies to Prevent and Advances in Purification Viral clearance studies are accomplished by operations of the VLP manufacturing process. Remove from Biologics Technologies challenging scaled-down versions of purification Barbara J. Potts, Ph.D., Senior Consultant, Potts and 12:00 pm Robust Virus Sponsored By steps with live viral “spikes”. These studies are Nelson Consulting, LLC Virus & Pathogen Clearance Filter Design Space via & Safety in Biologics typically conducted in BSL-2 facilities and are costly. Strategies will be presented to prevent the A non-infectious viral surrogate would be useful for Enhanced Macroscale contamination of biologics with porcine circovirus, ANALYTICAL & QUALITY scientists developing or characterizing downstream Surface Geometry porcine hepatitis E virus from contaminated purification process steps. Discussed here is an Host Cell Proteins: Detection, Aernout Martens, Ph.D., Global Product Manager, porcine raw materials and from mycoplasmas attempt to use a Virus Like Particle to determine Virus Filtration, Pall Life Sciences Analysis & Removal found in peptones and bovine sera. Removal and the Mouse Minute Virus removal efficacy of a Maximizing virus filter capacity while maintaining inactivation methods will be presented that have Early Analytical Development for chromatography column step. high viral safety is critical to overall bioprocess been successful including a case study of porcine Biotherapeutics cost of goods reduction. In order to provide both circovirus in pepsin. 10:00 Networking Coffee Break Process Characterization, high protein capacity and robust virus retention we have applied surface geometry to a virus filter Qualification & Control CLEARANCE SELECTION METHODS membrane at the macroscale. This enhances the 2:30 Future of Viral Clearance FORMULATION & STABILITY available surface area relative to the frontal surface Speaker to be Announced 10:30 Retrospective Evaluation of Low pH area. The improvement in capacity is demonstrated Rapid Methods to Assess With an increasing number of biological drug Viral Inactivation and Viral Filtration Data using colloidal gold, polyclonal IgG, and monoclonal producers outsourcing their viral clearance studies, Quality & Stability of Biologics from Multiple Company Collaboration antibody solutions. Bacteriophage data confirms exploring the relationship between CRO and biologic Overcoming Formulation Xinfang Li, Principal Scientist, Process Science and that keeping the downstream retentive surface of manufacturers is crucial to the success of viral Challenges Engineering, ImmunoGen, Inc. the membrane unchanged maintains the retentive clearance. characteristics of the membrane, improving the Considerable resources are spent within the • The role of biologics companies in downstream High-Concentration balance between critical performance parameters. biopharmaceutical industry to perform viral clearance studies Protein Formulations clearance studies which are conducted for widely 12:30 Luncheon Presentation (Sponsorship • Establish clear target clearance goals CELL & GENE THERAPY PRODUCTION used unit operations that are known to have robust and effective retrovirus clearance capability. Opportunity Available) or Lunch on Your Own Cell Therapy CMC, 3:30 Close of Conference The collaborative analysis from the members of 1:15 Session Break Quality & Analytics BioPhorum Development Group Viral Clearance Cell Therapy Bioproduction, Working Team considers two common virus FUTURE OF VIRAL CLEARANCE & Operations & Logistics reduction steps in biopharmaceutical processes: low pH viral inactivation and viral filtration. BIOLOGIC SAFETY Gene Therapy Bioproduction 11:00 Viral Clearance for Commercial-Scale 1:25 Chairperson’s Remarks Manufacturing & CMC Norovirus Virus-Like Particle Manufacturing David Cetlin, Founder & CEO, MockV Solutions LLC Strategies for Biologics Processes 1:30 Progress in Development of Virus- Ross Taylor, Ph.D., Director, Process Development, SPONSORS & EXHIBITORS Retentive Size-Exclusion Filter Paper Takeda Pharmaceuticals Inc. Albert Mihranyan, PhD, Professor of HOTEL & TRAVEL Norovirus infection is the most common cause REGISTRATION INFORMATION
BioprocessingSummit.com ◄ 27 ► COVER EVENT AT A GLANCE PLENARY KEYNOTE SESSION STREAM #3 SHORT COURSES TRAINING SEMINARS UPSTREAM PROCESSING Analytical & Quality Optimizing Cell Culture Technology Bioproduction: Scale, Bioreactors & Disposables The weeklong Analytical & Quality stream offers in-depth updates on critical steps in biopharmaceutical development that Optimizing Cell Line Development impact product quality, safety and regulatory compliance. Separate meetings will focus on the detection, analysis and removal DOWNSTREAM PROCESSING Continuous Processing in of host cell proteins, early stage analytical development and the implementation of process qualification and control strategies. Biopharm Manufacturing Over fifty in-depth presentations will give attendees insight into the best practices being applied across the industry. Advances in Purification Technologies Virus & Pathogen Clearance & Safety in Biologics ANALYTICAL & QUALITY Host Cell Proteins: Detection, Analysis & Removal Early Analytical Development for AUGUST 15-16 Biotherapeutics Process Characterization, Host Cell Proteins: Detection, Analysis & Removal Qualification & Control FORMULATION & STABILITY Rapid Methods to Assess AUGUST 17-18 Quality & Stability of Biologics Overcoming Formulation Early Analytical Development for Biotherapeutics Challenges High-Concentration Protein Formulations AUGUST 18-19 CELL & GENE THERAPY PRODUCTION Cell Therapy CMC, Process Characterization, Qualification & Control Quality & Analytics Cell Therapy Bioproduction, Operations & Logistics Gene Therapy Bioproduction Manufacturing & CMC Strategies for Biologics SPONSORS & EXHIBITORS HOTEL & TRAVEL REGISTRATION INFORMATION
BioprocessingSummit.com COVER Analytical & Quality EVENT AT A GLANCE Inaugural PLENARY KEYNOTE SESSION SHORT COURSES Host Cell Proteins TRAINING SEMINARS Detecting, Analyzing and Controlling HCPs for Improved Quality and Safety UPSTREAM PROCESSING Optimizing Cell Culture Technology Bioproduction: Scale, Bioreactors MONDAY, AUGUST 15 to illustrate the use of risk assessment for HCP commonly used as the calibration standards for & Disposables identification and control with consideration of HCP ELISA. The generation of mock HCPs often 8:00 am Short Course Registration clinical information. follows similar or identical upstream manufacturing Optimizing Cell Line Development process as the production cell culture. We explored 2:15 Immunogenicity Risk Assessment the upstream process range or design space where DOWNSTREAM PROCESSING 9:00 – 11:30 Recommended Morning Short Course* of Host Cell Protein in Therapeutic a platform assay can remain to be relevant and Continuous Processing in suitable for monitoring HCP clearance. New USP Initiatives and Standards for Biologics Monoclonal Antibodies Biopharm Manufacturing Qingchun Zhang, Ph.D., Senior Scientist, Amgen * Separate registration required, see page 5 for details. 4:30 Breakout Discussions Advances in Purification In this study, we investigated the HCP profiles for Technologies 11:30 Main Conference Registration Opens partially purified in-process samples and highly 5:30 Grand Opening Reception in the purified DS-like samples for four therapeutic mAbs. Exhibit Hall with Poster Viewing Virus & Pathogen Clearance CURRENT HCP CHALLENGES, RISK The results indicated in-process samples with & Safety in Biologics ASSESSMENT & CONTROL STRATEGIES up to 4000 ppm HCP content (levels >200 times 7:00 End of Day greater than the drug substance) did not pose a ANALYTICAL & QUALITY 1:00 pm Chairperson’s Opening Remarks higher immunogenicity risk than highly-purified DS samples. Furthermore, sequence associated TUESDAY, AUGUST 16 Host Cell Proteins: Detection, Judy Shimoni, Ph.D., Senior Scientist and Group immunogenicity risk of over 20 common HCPs was Analysis & Removal Leader, PTDU, Protein Analytical Chemistry, evaluated using in silico algorithms. 7:30 am Registration Opens and Morning Early Analytical Development for Genentech Coffee Biotherapeutics 2:45 Refreshment Break 1:10 KEYNOTE PRESENTATION: HCP CHARACTERISATION & MASS Process Characterization, Best Practices for Host Cell Protein Qualification & Control HCP RISK ASSESSMENT, CONTROL SPECTROMETRY STRATEGIES Measurement: USP Chapter <1132> STRATEGIES & DESIGN SPACE FORMULATION & STABILITY Maura Kibbey, Ph.D., Director, Science and 7:55 Chairperson’s Remarks Rapid Methods to Assess Standards, Global Biologics, USP 3:15 HCP Assays as In-Process Tests Phoebe Baldus, Ph.D., Principal Scientist and Group Quality & Stability of Biologics USP’s official chapter <1132> provides Carl Co, Ph.D., Senior Scientist, Biogen Idec Leader, Pfizer expert guidance on (1) HCP immunogen and HCPs must be removed to ensure product efficacy Overcoming Formulation standard preparation and characterization; (2) and safety. ICH specifications guidance, Q6B, states 8:00 Adding MS to a Host Cell Protein Challenges immunization; (3) purification and characterization that for certain impurities, testing of drug substance Workflow High-Concentration of antibodies; (4) immunoassay development may not be included in specifications if control Phoebe Baldus, Ph.D., Principal Scientist and Group and validation; (5) lifecycle management; and Protein Formulations or removal to acceptable levels is demonstrated. Leader, Pfizer (6) orthogonal tools to supplement the primary Case studies will be presented which highlight the CELL & GENE THERAPY PRODUCTION immunoassay technology. Due to these complex, challenges of our strategy of performing HCP assays 8:30 Analysis of Host Cell Proteins multi-antigen HCP immunoassays, emphasis for in-process and not for release. throughout Biopharmaceutical Purification Cell Therapy CMC, is placed on unique challenges (e.g., sample Quality & Analytics Martha Stapels, Ph.D., Senior Scientist, dilution linearity, validation, and reporting 3:45 Sponsored Presentation (Opportunity Biopharmaceuticals Development, Sanofi Genzyme Cell Therapy Bioproduction, of results). Available) Corporation Operations & Logistics Methods have been developed to identify and 4:00 Platform Reagents. The Design-Space Gene Therapy Bioproduction 1:45 HCP Testing, Control Strategy and Risk quantify HCPs by mass spectrometry, but these Assessment of Generating Platform Reagents for HCP typically involve 2D chromatography. We have Manufacturing & CMC Judy Shimoni, Ph.D., Senior Scientist and Group Monitoring by Assessing the Impact of developed a high throughput method to analyze Strategies for Biologics Leader, PTDU, Protein Analytical Chemistry, Upstream Process Condition Variations on HCPs where peptide identification occurs where the Genentech HCP Expression Profile HCP is most abundant, followed by quantitation in SPONSORS & EXHIBITORS The presentation is intended to share some current Fengqiang Wang, Ph.D., Associate Principal samples across the purification process. Statistical HOTEL & TRAVEL thinking and to use case studies to demonstrate the Scientist, Merck Research Laboratories, Merck & analysis is then used to evaluate purification steps application of orthogonal methods complementing Co. Inc. and optimize the process. REGISTRATION INFORMATION HCP workhorse testing method and supporting Mock host cell proteins produced from a null process development and control strategy; also cell line without the product-encoding gene are BioprocessingSummit.com ◄ 29 ► COVER Analytical & Quality EVENT AT A GLANCE Inaugural PLENARY KEYNOTE SESSION SHORT COURSES Host Cell Proteins TRAINING SEMINARS Detecting, Analyzing and Controlling HCPs for Improved Quality and Safety UPSTREAM PROCESSING Optimizing Cell Culture Technology Sponsored By Bioproduction: Scale, Bioreactors 9:00 Utilization of Mass Spectrometry for HCP TESTING STRATEGIES FOR 12:00 pm Integration of & Disposables HCP Analysis during Process Development BIOSIMILARS Data from Orthogonal Gang Xiao, Ph.D., Scientist, Process Development, Methods to Provide a Optimizing Cell Line Development Amgen 10:30 Quantitative MS/MS in the Comprehensive Analysis of HCPs in Final DOWNSTREAM PROCESSING It is critical to measure individual HCP by mass Determination of HCP Levels Drug Substance spectrometry in order to troubleshoot or optimize Continuous Processing in Nesredin A Mussa, Ph.D., Head, Process Analytics Kenneth Hoffman, Ph.D., President, Cygnus the bioprocess. In this presentation, we will Biopharm Manufacturing and Method Development, Biologics Development, Technologies demonstrate how mass spectrometry can be Global Manufacturing and Supply, Bristol-Myers Because of certain limitations of ELISA, orthogonal Advances in Purification utilized for HCP analysis during the bioprocess from Squibb methods of analysis are required. This talk discusses Technologies upstream cell production, through downstream how data from 2D HPLC, Antibody Affinity purification, to final product releasing. 11:00 Host Cell Protein Analysis by Mass Virus & Pathogen Clearance Extraction (AAE), 2D PAGE, and LCMS/MS when Spectrometry and Its Application in a integrated with ELISA data can be used to give a 9:30 HCP Identification and Sponsored By & Safety in Biologics Comparability Exercise comprehensive analysis of individual HCPs that Absolute Quantification with Michael Gattner, Ph.D., Associate Scientist, persist through a purification process. ANALYTICAL & QUALITY a Multifaceted Mass Physico-Chemical Characterization, Sandoz Sponsored By Host Cell Proteins: Detection, Spectrometry Platform Biopharmaceuticals/ Hexal AG 12:30 Reducing Operator Analysis & Removal Laura McIntosh, Director, Vice President, Host cell proteins (HCPs) are process-related Intervention with a Early Analytical Development for Translational Research, ProteoCarta Management, impurities present in biopharmaceuticals and are Comprehensive Approach to Biotherapeutics CAPRION generally considered to be critical quality attributes. HCP and Residual Protein A Testing A highly sensitive discovery platform has been Here we discuss the usage of ELISA and mass Renee Tobias, Product Manager, Pall ForteBio LLC Process Characterization, developed, employing fractionation, deglycosylation, spectrometry to monitor HCP populations in the Qualification & Control Using an automatable alternative to ELISAs for Host custom databases, and multiple search engines, to context of comparability and similarity excercises. Cell Protein and Residual Protein A detection could FORMULATION & STABILITY enable the identification and relative quantification A case study employing mass spectrometry-based significantly reduce the number of manual assay of low ppm HCP from any host cell (CHO, human, HCP analysis following a manufacturing change Rapid Methods to Assess steps – and the variability those steps introduce. The yeast, bacteria), without the need to remove the is provided. Ready BLI kits from Pall ForteBio provide a more Quality & Stability of Biologics drug substance. Targeted MRM mass spectrometry complete solution to impurity testing in bioprocess 11:30 Host Cell Proteins: The Hidden Side of Overcoming Formulation is then used for absolute quantification down development and in-process QC. to 1 ppm. These assays enable process Biosimilarity Assessment Challenges improvement, clearance monitoring, determination Roxana Butoi, Ph.D., Manager, Analytical 1:15 Dessert Refreshment Break in the High-Concentration of reproducibility, impact of scale-up and other Development, R&D Platform Technology and Exhibit Hall with Poster Viewing Protein Formulations monitoring needs for antibodies, blood products or Science, GlaxoSmithKline peptide manufacture. Results illustrate the efforts to assess CELL & GENE THERAPY PRODUCTION ASSAY DEVELOPMENT – PLATFORMS, “biosimilarity” for a specific group of process- Cell Therapy CMC, 9:45 Coffee Break in the Exhibit Hall with related impurities, the host cell proteins (HCP). VALIDATION, COVERAGE, CRITICAL Quality & Analytics Poster Viewing Extensive characterization of HCP in the drug REAGENTS Cell Therapy Bioproduction, substance of a biosimilar candidate guided process development to improve HCP clearance. The data 1:55 Chairperson’s Remarks Operations & Logistics presented illustrate the challenge of matching Fengqiang Wang, Ph.D., Associate Principal Gene Therapy Bioproduction the reference product on either quantitative or Scientist, Merck Research Laboratories, Merck & qualitative aspects of HCP impurities. Co. Inc. Manufacturing & CMC Strategies for Biologics 2:00 Platform Host Cell Protein Assays: How to Demonstrate Fitness for Use SPONSORS & EXHIBITORS Oliver Anderka, Ph.D., Fellow, Functional Lead HOTEL & TRAVEL REGISTRATION INFORMATION
BioprocessingSummit.com ◄ 30 ► COVER Analytical & Quality EVENT AT A GLANCE Inaugural PLENARY KEYNOTE SESSION SHORT COURSES Host Cell Proteins TRAINING SEMINARS Detecting, Analyzing and Controlling HCPs for Improved Quality and Safety UPSTREAM PROCESSING Bioanalytics, Novartis Pharma AG Optimizing Cell Culture Technology HCP DETECTION IN BIOPROCESSING “Platform” host cell protein assays are used by Bioproduction: Scale, Bioreactors biomanufacturers to test a variety of products 4:15 Finding Needles in a Haystack – & Disposables made from the same type of host organism. An Strategies and Tools for HCP Identification Optimizing Cell Line Development outline is given of how to demonstrate a platform In Biopharmaceuticals assay’s fitness for use. This includes reagent Xiaohui Lu, Ph.D., Senior Scientist, BioPharma DOWNSTREAM PROCESSING characterization/qualification, reagent replacement, Development, Biogen assay validation, as well as criteria for application of a Continuous Processing in Identification of host cell proteins (HCPs) in platform assay to a new product or process. Biopharm Manufacturing biopharmaceuticals drug is hampered by the 2:30 HCP Analysis for a Spectrum of presence of overwhelming amount of the products. Advances in Purification This case study compared different strategies to Products Generated from Multiple CHO Technologies remove product and enrich HCPs. Applying these Cell Lines Including a Comparison of the Virus & Pathogen Clearance enrichment technologies will greatly improve Performance of a Panel of Different HCP & Safety in Biologics MS-based HCP identification, and provide much Commercial Kits needed HCP information for purification process ANALYTICAL & QUALITY Girija Krishnamurthy, Ph.D., Director, Method development and risk assessments. Development, Bristol-Myers Squibb Host Cell Proteins: Detection, 4:45 Panel Session: Conference Wrap Up Analysis & Removal 3:00 Cover Your Assay: Approaches to Early Analytical Development for Evaluate HCP ELISA Reagent Coverage 5:15 End of Conference Biotherapeutics Denise Krawitz, Ph.D., Senior Manager Analytical 5:15 Registration for Dinner Short Course Operations, Genentech Process Characterization, In order to use ELISAs, we must demonstrate that Qualification & Control the antibodies used in the assay recognize a majority FORMULATION & STABILITY of the proteins produced by the host cell. While 2D SDS-PAGE and Western blots techniques are Rapid Methods to Assess valuable for a qualitative assessment of antibody Quality & Stability of Biologics coverage, they have limited value as a quantitative Overcoming Formulation tool. Additionally, platform HCP ELISAs are used Challenges to monitor HCPs in multiple products. Proteomics studies of different cell lines grown under different High-Concentration culture conditions suggest that the vast majority of Protein Formulations proteins expressed are the same between cell lines.
CELL & GENE THERAPY PRODUCTION 3:30 Refreshment Break in the Exhibit Hall Cell Therapy CMC, with Poster Viewing Quality & Analytics Cell Therapy Bioproduction, Operations & Logistics Gene Therapy Bioproduction Manufacturing & CMC Strategies for Biologics SPONSORS & EXHIBITORS HOTEL & TRAVEL REGISTRATION INFORMATION
BioprocessingSummit.com ◄ 31 ► COVER Analytical & Quality EVENT AT A GLANCE 4th Annual PLENARY KEYNOTE SESSION SHORT COURSES Early Analytical Development for Biotherapeutics TRAINING SEMINARS Optimizing the Selection and Performance of Preclinical Analytical Studies UPSTREAM PROCESSING Optimizing Cell Culture Technology WEDNESDAY, AUGUST 17 9:30 Best Practices for Early Stage Bioassay peptides with a wide range of molecular weight, Bioproduction: Scale, Bioreactors Development polarity and charge were studied. & Disposables 7:00 am Registration Opens and Morning Kari Sweeney Efferen, Ph.D, Principal Scientist, 11:45 Increasing Analytical Throughput Optimizing Cell Line Development Coffee Pfizer for Late Phase Process Development - Incorporation of bioassays is crucial in bioprocess DOWNSTREAM PROCESSING 8:05 Chairperson’s Opening Remarks and formulation development since it is often An Automation Approach for Sample Continuous Processing in Jason Huang, Ph.D., Senior Research Investigator, difficult to assess the relevant structure or critical Management Biopharm Manufacturing Bristol-Myers Squibb epitopes linked to a particular biological activity William Grimm, Associate Scientist, Process using traditional biochemical or biophysical Development & Analytics, Bristol-Myers Squibb Advances in Purification 8:15 KEYNOTE PRESENTATION: techniques during early development. It is A liquid handling system has been configured to act Technologies Integrated Microfluidic Capillary necessary to ensure that the assay answers the as a sample transfer and dilution platform that can Virus & Pathogen Clearance Electrophoresis-Electrospray Devices for question being asked, such as stability of a critical dynamically organize samples on 96-well plates. & Safety in Biologics Analysis of Intact mAbs and ADCs epitope or relevant structure that can be linked to Automation of ELISA assay setup is handled by J. Michael Ramsey, Ph.D., Minnie N. Goldby particular biological activity of that material. a combination of software scripts and Excel VBA ANALYTICAL & QUALITY commands, this allows for a simple spreadsheet Distinguished Professor of Chemistry, University 10:00 Coffee Break in the Exhibit Hall with Host Cell Proteins: Detection, of North Carolina to be used by the analyst to setup a run. Routine Poster Viewing use of this technology for sample preparation Microfluidic devices that include monolithically Analysis & Removal and organization is invaluable for projects in an integrated nano-electrospray emitters are Early Analytical Development for 10:45 Intact Mass Analysis of Monoclonal accelerated drug development timeline. used to electrophoretically separate charge Antibodies by Capillary Electrophoresis Biotherapeutics variants of intact monoclonal antibody (mAbs) (CE) – Mass Spectrometry (MS) 12:15 Luncheon Presentation (Sponsorship biotherapeutics and antibody drug conjugates Process Characterization, Opportunity Available) or Lunch on Your Own (ADCs). Specialized channel wall coatings Mei Han, Scientist, Pharmacokinetics & Drug Qualification & Control Metabolism, Amgen, Inc. allow the use of electrospray friendly buffer 1:00 Session Break FORMULATION & STABILITY systems while maintaining high performance Intact mass protein characterization provides important sequence integrity and post-translational Rapid Methods to Assess electrophoretic separative performance. Analyses modification information. While LC-MS is widely HIGH THROUGHPUT ANALYTICAL Quality & Stability of Biologics are typically performed in three minutes with little sample preparation while utilizing mass used in the biopharmaceutical industry, CE-MS DEVELOPMENT Overcoming Formulation spectrometers with modest resolving power. is an attractive alternative for its high separation Challenges efficiency, sensitivity, and minimal sample carryover. 1:45 Chairperson’s Remarks ASSAY DEVELOPMENT However, the intrinsic technical difficulty has Udayanath Aich, Principal Scientist, Process High-Concentration hindered the application of this technique. Here, we Analytics, Biopharmaceutical Development, Sanofi- Protein Formulations 9:00 Characterization of Therapeutic Protein report the implementation of a recently developed Genzyme CELL & GENE THERAPY PRODUCTION by Two Dimensional-LC (2D-LC) System CE-MS interface for proteins from predose and postdose pharmacokinetic samples characterization. 1:50 Application of DoE to Optimize Cell Therapy CMC, Shenjiang Yu, Ph.D., Associate Principal Scientist, Product and Process Parameters Analytical Method Development, Merck Quality & Analytics 11:15 Ion-Pairing Effect on the Behavior Steve LaBrenz, Ph.D., Scientific Director, Janssen Two-dimensional liquid chromatography (2D-LC) is a of Synthetic Peptides in Reversed-Phase R&D LLC Cell Therapy Bioproduction, powerful tool for analyzing highly complex samples Liquid Chromatography The adoption of Quality by Design during drug Operations & Logistics such as therapeutic proteins. Both dimensions product development as a regulatory requirement have orthogonal separation selectivity, thereby Jason Z. Huang, Ph.D., Senior Research Investigator, Gene Therapy Bioproduction may necessitate the use of DoE in the laboratory to increasing the potential peak capacity to the Bristol-Myers Squibb satisfy development activities. Multivariate analysis product of the individual peak capacities. With this We have conducted a systematic study of ion Manufacturing & CMC can be accomplished in many different experimental advanced technology, 2D-LC provides the possibility pairing reagents for the reversed-phase HPLC Strategies for Biologics designs (Taguchi, traditional, definitive), as well as to combine two orthogonal methods and discover analysis of therapeutic peptides with the goal of modeling approaches (PLS, PCA). To gain the full SPONSORS & EXHIBITORS the correlation of the data. This correlation provides improving impurity separations. We studied the benefit of a DoE, consideration needs to be given to critical information for total characterization of influence of ion pairing reagents TFA, NaClO4 and HOTEL & TRAVEL KPF6 on retention and peak shape of peptides on the normalized form of data from which means and therapeutic proteins. limits can be derived. REGISTRATION INFORMATION RPLC. As a model system several cyclic and linear
BioprocessingSummit.com ◄ 32 ► COVER Analytical & Quality EVENT AT A GLANCE 4th Annual PLENARY KEYNOTE SESSION SHORT COURSES Early Analytical Development for Biotherapeutics TRAINING SEMINARS Optimizing the Selection and Performance of Preclinical Analytical Studies UPSTREAM PROCESSING Optimizing Cell Culture Technology 2:20 Recent Advances in Process Analytics achieve this it is necessary to analyze many samples 9:00 Mass Spectrometric Analysis of Novel Bioproduction: Scale, Bioreactors for High-throughput Analysis of N-Glycans in a short time, therefore HT analytics is often Antibody-Drug Conjugates (ADCs) in a Fast- & Disposables from Therapeutic Recombinant Proteins applied in early development. Microchip capillary Paced Environment electrophoresis is one of the HT techniques with Optimizing Cell Line Development Udayanath Aich, Principal Scientist, Process Lintao Wang, Ph.D., Principal Scientist and many advantages but also some disadvantages, Analytics, Biopharmaceutical Development, Sanofi- Mass Spectrometry Group Lead, Analytical and which will be presented in this talk. DOWNSTREAM PROCESSING Genzyme Pharmaceutical Science, ImmunoGen, Inc. Continuous Processing in With the increase of regulatory expectations, the 3:50 Refreshment Break in the Exhibit Hall Designing a safe and effective ADC involves Biopharm Manufacturing evolution of PAT and emerging manufacturing with Poster Viewing extensive screening of various antibody, linker Advances in Purification processes create a demand for robust, sensitive, and payload combinations, which require efficient accurate profiling of protein glycosylation. Potential analysis of a large number of samples with different Technologies 4:45 Plenary Keynote Session (see page 3 shortcomings in protein glycosylation profiling for details) physicochemical properties. This presentation Virus & Pathogen Clearance include the high hands-on time required for sample will focus on the capabilities of size-exclusion & Safety in Biologics preparation and several hours for data acquisition 6:00 Networking Reception in the Exhibit chromatography coupled with electrospray mass and analysis. This presentation will focus on the Hall with Poster Viewing spectrometry (SEC-MS), a platform assay that is ANALYTICAL & QUALITY state-of-art process analytics for the high throughput used for generating important information about Host Cell Proteins: Detection, sample preparation and analysis of N-glyans from 7:00 End of Day quality attributes of both antibodies and ADCs in a Analysis & Removal therapeutic proteins. fast-paced developmental environment. Early Analytical Development for 2:50 High Throughput Assays for Evaluation THURSDAY, AUGUST 18 9:30 A Combination of Structural and Biotherapeutics of Post-Translational Modifications Empirical Analyses Delineates the Key 8:00 am Registration Opens and Morning Andras Guttman, Ph.D., Senior Manager, SCIEX; Contacts Mediating Stability and Affinity Process Characterization, Coffee Qualification & Control MTA-PE Lendulet Professor of Translational Increases in an Optimized Biotherapeutic Glycomics, Horváth Csaba Laboratory of ANALYTICAL DEVELOPMENT FOR Single-Chain Fv FORMULATION & STABILITY Bioseparation Sciences, University of Debrecen, Joel Bard, Ph.D., Principal Research Scientist, Pfizer Hungary Rapid Methods to Assess NOVEL BIOTHERAPEUTICS Single-chain Fv proteins are key building blocks of With the sharp increase in the number of Quality & Stability of Biologics 8:25 Chairperson’s Remarks bispecific therapeutic antibodies. The causes of approved protein therapeutics, comprehensive scFv instability problems remain poorly understood. Joel Bard, Ph.D., Principal Research Scientist, Pfizer Overcoming Formulation characterization of these new generation drugs We performed detailed structural and biochemical Challenges is crucial for the biopharmaceutical industry and analyses to understand the mechanisms that confer regulatory agencies. Biotherapeutics possess 8:30 Analytics by Design: Early Stage High-Concentration Method Development Guided by Molecular both tighter binding and improved stability on the various post-translational modifications (PTMs) result of a phage display optimization campaign. Protein Formulations Features and prone to potential degradation hotspots during This work demonstrates that, aside from being the CELL & GENE THERAPY PRODUCTION the manufacturing process that may affect their Felix Schumacher, Ph.D., Senior Scientist, Large critical mediators of target binding, CDRs may also efficacy and immunogenicity. This presentation Molecule Research, Roche Diagnostics GmbH, be primary drivers of biotherapeutic developability. Cell Therapy CMC, will cover the state of the art of high throughput Germany Quality & Analytics separation methods for structural elucidation of “Analytics by Design” is a novel concept for early 10:00 Coffee Break in the Exhibit Hall with Cell Therapy Bioproduction, protein modifications. stage analytical method development. Here, Poster Viewing Operations & Logistics analytical packages are tailored to the critical quality 3:20 Application of HT Electrophoresis in attributes of next-generation biotherapeutics, in Gene Therapy Bioproduction Early Biosimilar Development contrast to the use of generic platform methods. Urška Kristan, Ph.D., Researcher and A streamlined method development is based on Manufacturing & CMC Analyst, Analytical Development, Sandoz theoretical considerations and data collected during Strategies for Biologics Biopharmaceuticals, Slovenia developability assessment. Such an approach yields SPONSORS & EXHIBITORS Current initiatives in biosimilar development require valuable insights into multiple aspects of early thorough understanding of the relationship between stage process development, which may guide CMC HOTEL & TRAVEL product quality and process parameters. In order to considerations of future project stages. REGISTRATION INFORMATION
BioprocessingSummit.com ◄ 33 ► COVER Analytical & Quality EVENT AT A GLANCE 4th Annual PLENARY KEYNOTE SESSION SHORT COURSES Early Analytical Development for Biotherapeutics TRAINING SEMINARS Optimizing the Selection and Performance of Preclinical Analytical Studies UPSTREAM PROCESSING Optimizing Cell Culture Technology 10:45 Characterization of Formulation Development, Regeneron Pharmaceuticals, Inc. Bioproduction: Scale, Bioreactors Excipient Using Thermal and Spectroscopic Developing a method to separate and quantify individual mAb’s from a mixture of multiple & Disposables Methods for Robust Drug Product Development monoclonal antibodies is challenging because the Optimizing Cell Line Development mAb molecules may have similar molecular weights, Rushikesh Patel, Associate Research Scientist, protein structures and molecular charges. In this Bristol-Myers Squibb DOWNSTREAM PROCESSING case study, we present our approaches to developing Continuous Processing in Excipient performance during processing can have a quantification method for a co-formulation of three a significant impact on critical quality attributes of Biopharm Manufacturing different monoclonal antibodies. In addition, the the product. Therefore, detailed characterization of advantages and limitations of the different methods Advances in Purification excipient behavior is critical during early development will be discussed. Technologies and process design. Here, we present application of multiple thermal and spectroscopic methods Virus & Pathogen Clearance 12:15 Lunch Available for Purchase in the for understanding phase behavior of trehalose, Exhibit Hall & Safety in Biologics a commonly used stabilizer, during freeze/thaw process. The impact of phase change on product ANALYTICAL & QUALITY 1:15 Dessert Refreshment Break in the quality of a fusion protein is also highlighted. Exhibit Hall with Poster Viewing Host Cell Proteins: Detection, Analysis & Removal 11:15 A Novel Approach to Isolate and 1:55 End of Conference Characterize Product Variants in a Early Analytical Development for Monoclonal Antibody Biotherapeutics Xiaoxiao Li, Ph.D., Senior Scientist, Analytical Process Characterization, Development, Abbvie Biotherapeutics Qualification & Control Characterization of binding potency of product variants is required as part of the well-characterized FORMULATION & STABILITY biotherapeutic paradigm. Here we present a novel Rapid Methods to Assess “divide and conquer” approach to directly isolate Quality & Stability of Biologics variants that differ in binding potency. This approach Overcoming Formulation is applicable to a wide range of biotherapeutics, requires no prior knowledge of the variants, and Challenges enables rapid identification and characterization of High-Concentration all variants that differ in binding potency from the Protein Formulations main product. CELL & GENE THERAPY PRODUCTION 11:45 A Chromatographic Method for Cell Therapy CMC, Quantifying Individual Monoclonal Quality & Analytics Antibodies (mAb) in a Co-Formulated Solution Cell Therapy Bioproduction, Lin Luo, Research Associate, Formulation Operations & Logistics Gene Therapy Bioproduction Manufacturing & CMC Strategies for Biologics SPONSORS & EXHIBITORS HOTEL & TRAVEL REGISTRATION INFORMATION
BioprocessingSummit.com ◄ 34 ► COVER Analytical & Quality EVENT AT A GLANCE 2nd Annual PLENARY KEYNOTE SESSION SHORT COURSES Process Characterization, Qualification and Control TRAINING SEMINARS A best practices forum for process characterization, control strategies and maintaining UPSTREAM PROCESSING quality throughout the product lifecycle Optimizing Cell Culture Technology Bioproduction: Scale, Bioreactors THURSDAY, AUGUST 18 the most critical parameters for release assays for “standard” analytical methods reducing analytical & Disposables each new molecule, saving a more comprehensive testing time and allowing for more comprehensive Optimizing Cell Line Development 11:30 am Registration Opens robustness study for later in development. and timely support of process characterization and development. DOWNSTREAM PROCESSING 12:15 pm Lunch Available for Purchase in 3:15 Efficient High-Throughput Biological Continuous Processing in the Exhibit Hall Process Characterization 4:45 SPEAKER HAS CANCELLED. Biopharm Manufacturing Mitchell Tai, Ph.D., Scientist, Biologics Process DELEGATES MAY ATTEND SESSIONS IN 1:15 Dessert Refreshment Break in the Development, Bristol-Myers Squibb ANY OF THE OTHER STREAMS AT THIS Advances in Purification Exhibit Hall with Poster Viewing Systems for high-throughput process TIME. Technologies characterization are poised to greatly improve 1:55 Chairperson’s Opening Remarks Virus & Pathogen Clearance development timelines. Here we combine Analytical Considerations for QbD and Paul Bigwarfe, Ph.D., Director, Analytical Sciences, & Safety in Biologics the Sartorius ambr250 system with definitive PAT of Biotechnology/Biosimilar Process Regeneron Pharmaceuticals screening design experiments to perform rapid Development ANALYTICAL & QUALITY process characterization for a microbial process Nadine M. Ritter, Ph.D., President & Principal 2:00 KEYNOTE PRESENTATION: Line of Host Cell Proteins: Detection, for recombinant protein production. Main effects Advisor, Global Biotech Experts LLC Sight to the Process Control Strategy: A Analysis & Removal screening and process modeling can be generated The value derived from QbD studies and PAT Plan for Commercial Success within a single round of experimentation. The results measurements is only as good as the analytical Early Analytical Development for Timothy Schofield, Senior Advisor, demonstrate a quality-by-design (QbD) approach methods they employ. If analytical data are Biotherapeutics GlaxoSmithKline for both early-stage process development and late- inaccurate or inconsistent due to method Process Characterization, The biopharmaceutical control strategy is a stage process characterization. performance capabilities, the observations and covenant between the manufacturer and the conclusions could be erroneous. This presentation Qualification & Control 3:45 Considerations for Sponsored by customer. Early consideration of manufacturing will highlight some of the most challenging analytical FORMULATION & STABILITY conditions and constraints, coupled with Material Qualification aspects of process characterization, illustrate During Process Development Rapid Methods to Assess process and analytical knowledge, inform examples of common mistakes made, and provide Quality & Stability of Biologics process and analytical development. The Stephen Doherty, Ph.D., Department Head, recommendations on how to minimize the analytical control strategy represents the translation Analytical Chemistry, Toxikon Corporation risks to process design and process control. Overcoming Formulation of development experiences and supporting This presentation will outline key considerations Challenges plans to ensure product quality and supply. This when selecting material and components for 5:15 End of Day presentation highlights a proactive approach single use systems. Thought processes and High-Concentration 5:15 Registration for Dinner Short Course to development of a robust control strategy evaluation strategies related to the selection of Protein Formulations that meets the requirements of both patients these materials and components will be discussed. CELL & GENE THERAPY PRODUCTION and manufacturers. This presentation will benefit scientists and FRIDAY, AUGUST 19 engineers engaged in the development, production Cell Therapy CMC, and implementation of single use systems in 8:00 am Registration Opens and Morning Quality & Analytics PROCESS CHARACTERIZATION AND understanding in the selection of these materials. Coffee Cell Therapy Bioproduction, DESIGN SPACE 4:00 Refreshment Break Operations & Logistics 2:45 DOE Studies in Support of Early Stage PROCESS QUALIFICATION AND Gene Therapy Bioproduction Process Characterization 4:15 Multi-Attribute LC/MS Methods for VALIDATION Paul Bigwarfe, Ph.D., Director, Analytical Sciences, Process Characterization and Design Space Manufacturing & CMC Industrial Operations and Product Supply, Development 8:25 Chairperson’s Remarks Strategies for Biologics Regeneron Pharmaceuticals Kristin Krukenberg, Ph.D., Analytical Scientist, Stephan O. Krause, Ph.D., Director, QA Technical Support, AstraZeneca Biologics SPONSORS & EXHIBITORS A DOE is becoming an essential part of the method Process Development, Shire development life cycle to better understand the Complex biologics provide a unique analytical 8:30 Continuous Process Validation HOTEL & TRAVEL acceptable ranges for the method. However, for challenge for characterizing and monitoring multiple Stephan O. Krause, Ph.D., Director, QA Technical methods at preclinical and Phase I stages, resources CQAs. We developed an LC/MS based approach REGISTRATION INFORMATION Support, AstraZeneca Biologics may not exist to evaluate every parameter. A that provides more detailed information than six rational strategy has been designed to analyze BioprocessingSummit.com ◄ 35 ► COVER EVENT AT A GLANCE Analytical & Quality 2nd Annual PLENARY KEYNOTE SESSION SHORT COURSES Process Characterization, Qualification and Control TRAINING SEMINARS A best practices forum for process characterization, control strategies and maintaining quality UPSTREAM PROCESSING Optimizing Cell Culture Technology throughout the product lifecycle Bioproduction: Scale, Bioreactors & Disposables 9:00 Extraction Study of a Cartridge Filter 11:00 Plant-Wide Process Control Model for in Bio Manufacturing : Regulatory, industry and Optimizing Cell Line Development Based upon the BioPhorum Operations Biologics Manufacturing risk considerations for designing a successful Group (BPOG) Recommendation Richard Braatz, Ph.D., Edwin R. Gilliland Professor extractables leachables program. A review of current regulatory environment, a summary of DOWNSTREAM PROCESSING Kurt Moyer, Ph.D., Director, Research, NSF Health of Chemical Engineering, Massachusetts Institute of recognized industry schools of thought and risk Continuous Processing in Sciences Technology to be considered in development of a customized A case study is presented in which mechanistic Biopharm Manufacturing In this study the extractables from a process filter approach that works for your system and models are constructed for all unit operations in an Advances in Purification were evaluated based upon the protocol developed product application. by BPOG. The sample was extracted in six extraction integrated biopharmaceutical manufacturing pilot Technologies solvents at 40°C with orbital rotation for 7 days. plant. The individual models are interconnected to 1:15 Session Break Virus & Pathogen Clearance Sample extracts were analyzed for organic and form a dynamic operations model of the entire plant. & Safety in Biologics inorganic extractables. For the organic extractables, The mechanistic models are used in the design of a total of 9 were observed with 6 being identified. control systems for individual unit operations, and a ANALYTICAL & QUALITY For the inorganic extractables, a total of 7 were strategy is described in which the overall plantwide Host Cell Proteins: Detection, observed and identified. control strategy is designed to suppress the effects of disturbances and uncertainties on the critical Analysis & Removal 9:30 Whole Molecule LC-MS Analysis for quality attributes. Early Analytical Development for HT Monitoring of Asn-Ser Substitution Biotherapeutics in an Antibody: Challenges in Method 11:30 Production Process Optimization and the Development of In-Process Process Characterization, Performance Assessment Controls Utilizing In-Line Spectroscopic Qualification & Control Céline Duhot, Assistant Manager, Biotech Process Sciences, Merck KGaA, Germany Measurements FORMULATION & STABILITY Product quality testing along process development Terrence Dobrowsky, Ph.D., Senior Engineer, Biogen Rapid Methods to Assess and characterization requires HT tools as well as Second generation process development offers an Quality & Stability of Biologics deep method understanding. This presentation opportunity to increase productivity and process discusses the case study of Asn-Ser substitution robustness by implementing process changes and Overcoming Formulation analysis of an IgG by whole molecule LC-MS. new control schemes. DOE practices were used Challenges Method performances were evaluated using a to optimize parameters and increase productivity High-Concentration genomically designed fully substituted antibody. Talk for an IgG producing CHO culture. Additionally, Raman spectroscopy enabled online glucose Protein Formulations addresses challenges in assessing semi-quantitative performances of MaxEntropy deconvoluted data for control to reduce glycated product. This work CELL & GENE THERAPY PRODUCTION the monitoring of small Dmass, not fully resolved increased productivity, decreased scale up and raw material risk, and demonstrated the relative ease of even on a state-of-the-art Q-Tof. Cell Therapy CMC, implementing advanced process analytics. Quality & Analytics 10:00 Networking Coffee Break Cell Therapy Bioproduction, 12:00 pm Application of a QbD Approach Operations & Logistics CONTROL STRATEGIES AND REAL to Control System Design for a Late Stage Monoclonal Antibody Gene Therapy Bioproduction TIME PROCESS CONTROL John Eschelbach, Ph.D., Scientist, Genentech Manufacturing & CMC 10:30 Technology Update: Process Analytical 12:30 Extractables Leachables Sponsored by Strategies for Biologics Technologies for Biologics Manufacturing Sheila G. Magil, Ph.D., Senior Consultant, for Single Use Systems in Bio SPONSORS & EXHIBITORS BioProcess Technology Consultants, Inc. Manufacturing HOTEL & TRAVEL Sandi Schaible, Directo,r Analytical Chemistry, WuXi AppTec REGISTRATION INFORMATION Extractables Leachables for Single Use Systems
BioprocessingSummit.com ◄ 36 ► COVER EVENT AT A GLANCE Analytical & Quality 2nd Annual PLENARY KEYNOTE SESSION SHORT COURSES Process Characterization, Qualification and Control TRAINING SEMINARS A best practices forum for process characterization, control strategies and maintaining quality UPSTREAM PROCESSING Optimizing Cell Culture Technology throughout the product lifecycle Bioproduction: Scale, Bioreactors 1:25 Chairperson’s Remarks 2:30 Application of Monte Carlo & Disposables Vincent Lau, Manager, Cell Culture Development, Simulations in the Development of Optimizing Cell Line Development Biogen Advanced Control Strategies and Manufacturing Facility Fit DOWNSTREAM PROCESSING 1:30 A Two-Tiered Approach to Process Vincent Lau, Manager, Cell Culture Development, Continuous Processing in Characterization for Late Stage Monoclonal Biogen Biopharm Manufacturing Antibody Programs Real time control strategies introduce additional Advances in Purification Michael Clark, Ph.D., Senior Scientist, Process complexity to the task of setting process controls on Technologies Sciences, AbbVie bioprocesses. Stochastic cell growth modeling and For efficiency of downstream process Monte Carlo simulation methods were evaluated Virus & Pathogen Clearance characterization, first tier DOE studies utilized for use in dealing with the relative complexity of & Safety in Biologics Definitive Screening Design (DSD) to identify the setting appropriate control limits for these types process parameters with the highest impact on ANALYTICAL & QUALITY of processes, as well as other common facility fit product quality attributes. This allowed minimalized or scale-up concerns. Use of these new methods Host Cell Proteins: Detection, second tier DOE studies using Response Surface allows for controls that ensure process consistency Analysis & Removal Design (RSD) to thoroughly investigate these high and reliable performance. impact parameters. This presentation describes the Early Analytical Development for statistical design and model selection of the studies, 3:00 Real-Time Product Attribute Control of Biotherapeutics and provides a comparative analysis of the DSD and N-Linked Glycan Levels Process Characterization, RSD results. Jeff Underhill, Senior Associate, Pivotal Cell Qualification & Control Sciences & Technology, Amgen 2:00 Vaccine Control Strategy Evolution The active control of product quality in biologic FORMULATION & STABILITY during Late-Stage Development and manufacturing processes is challenging due to Rapid Methods to Assess Commercialization measurement lags, system nonlinearities, and a lack Quality & Stability of Biologics Jennifer Dashnau, Ph.D., Director, Global Vaccines of robust control levers. A product attribute control and Biologics Commercialization, Merck & Co., Inc. Overcoming Formulation method utilizing nonlinear model predictive control Modern vaccines are amenable to characterization coupled with a rapid PAT system will be presented Challenges through a wide range of analytical techniques along with data from a pilot scale bioreactor run High-Concentration and robust control strategies may be developed. where this method successfully maintained a critical Protein Formulations GARDASIL®9 provides an opportunity to understand product quality attribute, percent high mannose, at a analytical control strategy evolution for a well- predetermined level. CELL & GENE THERAPY PRODUCTION characterized, recombinant vaccine product from Cell Therapy CMC, late-stage development to commercialization. 3:30 Close of Conference Quality & Analytics Examples of the impact of 1) process modifications made during development and 2) increased process Cell Therapy Bioproduction, and product performance understanding gained Operations & Logistics during commercialization on control strategy will Gene Therapy Bioproduction be provided. Manufacturing & CMC Strategies for Biologics SPONSORS & EXHIBITORS HOTEL & TRAVEL REGISTRATION INFORMATION
BioprocessingSummit.com ◄ 37 ► COVER EVENT AT A GLANCE PLENARY KEYNOTE SESSION STREAM #4 SHORT COURSES TRAINING SEMINARS UPSTREAM PROCESSING Formulation & Stability Optimizing Cell Culture Technology Bioproduction: Scale, Bioreactors & Disposables The weeklong Formulation & Stability stream brings together researchers in analytical and formulation sciences to share Optimizing Cell Line Development practical insights and case studies on biotherapeutic formulation development, development of high concentration protein DOWNSTREAM PROCESSING Continuous Processing in formulations, application of high-throughput/high resolution screening tools in early stage development and prediction and Biopharm Manufacturing manipulation for protein stability and instabilities. The three conferences together will feature cutting edge approaches for Advances in Purification Technologies understanding and managing protein formulation development, aggregation, high viscosity, and rapid analytical methods to Virus & Pathogen Clearance predict and screen formulation stability and quality issues. & Safety in Biologics ANALYTICAL & QUALITY Host Cell Proteins: Detection, Analysis & Removal Early Analytical Development for AUGUST 15-16 Biotherapeutics Process Characterization, Rapid Methods to Assess Quality & Stability of Biologics Qualification & Control FORMULATION & STABILITY Rapid Methods to Assess AUGUST 17-18 Quality & Stability of Biologics Overcoming Formulation Overcoming Formulation Challenges Challenges High-Concentration Protein Formulations AUGUST 18-19 CELL & GENE THERAPY PRODUCTION Cell Therapy CMC, High-Concentration Protein Formulations Quality & Analytics Cell Therapy Bioproduction, Operations & Logistics Gene Therapy Bioproduction Manufacturing & CMC Strategies for Biologics SPONSORS & EXHIBITORS HOTEL & TRAVEL REGISTRATION INFORMATION
BioprocessingSummit.com COVER Formulation & Stability EVENT AT A GLANCE 4th Annual PLENARY KEYNOTE SESSION SHORT COURSES Rapid Methods to Assess Quality & Stability of Biologics TRAINING SEMINARS Improving Prediction and Screening UPSTREAM PROCESSING Optimizing Cell Culture Technology Sponsored by Bioproduction: Scale, Bioreactors MONDAY, AUGUST 15 1:45 A Fast Method for Monitoring Multiple 3:45 For the First Time, & Disposables Attributes of Therapeutic Proteins Simultaneous Detection 8:00 am Short Course Registration Wei Xu, Ph.D., Principal Scientist, Analytical Method of Protein Aggregation and Affinity Optimizing Cell Line Development Development, Merck Measurements in a Single SPR Experiment 9:00 – 11:30 Recommended Morning DOWNSTREAM PROCESSING Therapeutic proteins such as mAbs have become Eric Reese, Ph.D., VP, Sales & Marketing, SensiQ Short Course* Continuous Processing in the top choice in treating many life threatening Technologies Inc Accelerated Stability Testing of Biologics disease conditions. An increased demand for Biopharm Manufacturing No SPR-biosensor has been capable of delivering * Separate registration required, see page 5 for details. new therapeutic mAbs and higher regulatory information on drug affinity and detect protein Advances in Purification expectations require the biopharmaceutical aggregates simultaneously in the same experiment. Technologies 11:30 Main Conference Registration Opens industry to increase efficacy in the research and SensiQ Technologies presents data from a current manufacturing process. High speed analytical Virus & Pathogen Clearance collaboration describing how this limitation can RAPID TOOLS FOR PREDICTION methods are highly desirable to speed up the be eliminated via simultaneous detection of both & Safety in Biologics bioprocess of therapeutic proteins. Discussion will protein aggregation and affinity determination in a & ASSESSMENT OF QUALITY & focus on development of a fast peptide mapping ANALYTICAL & QUALITY single experiment as enabled by Pioneer FE SPR. STABILITY method with very short sample preparation and Host Cell Proteins: Detection, analysis time using design of experiments. 4:00 Preclinical Tools in Formulation Analysis & Removal 1:00 pm Chairperson’s Opening Remarks Optimization to Improve Biological 2:15 Thermodynamics of Liquid-Liquid Early Analytical Development for Peter Tessier, Ph.D., Department of Chemical & Performance of Antibodies Biological Engineering, Center for Biotechnology Phase Separation in Monoclonal Antibody Biotherapeutics Sabine Eichling, Ph.D. Candidate, NBE Formulation and Interdisciplinary Studies, Rensselaer Polytechnic (mAb) Solutions Development, AbbVie Process Characterization, Institute Prasad Sarangapani, Ph.D., Scientist, Renegeron, Inc. Formulation development for biologics has focused Qualification & Control 2:45 Refreshment Break on the refinement of physical and chemical stability 1:10 KEYNOTE PRESENTATION: during shelf-live. Effects of formulation composition FORMULATION & STABILITY Methods for Selecting and Engineering 3:15 Rapid Peptide Mapping for Monitoring following s.c. injection were usually not considered. Rapid Methods to Assess Monoclonal Antibodies with Improved Multiple mAb Attributes The addition of biological read out parameters, such Quality & Stability of Biologics Biophysical Properties as bioavailability and ultimately efficacy, enables Nisana Andersen, Ph.D., Associate Scientist, Protein further improvement in the antibody transport to Overcoming Formulation Peter Tessier, Ph.D., Department of Chemical & Analytical Chemistry, Genentech, a Member of the the target tissue. The aim of this work is to develop Challenges Biological Engineering, Center for Biotechnology Roche Group and Interdisciplinary Studies, Rensselaer translatable models to understand the transport of Characterization of mAbs and their attributes is High-Concentration Polytechnic Institute antibodies following s.c. injection and predict the Protein Formulations an important aspect in identification of critical effect of the formulation. It is critical to optimize the biophysical properties quality attributes. LC-MS/MS peptide mapping, CELL & GENE THERAPY PRODUCTION of monoclonal antibodies in addition to their intended for in-depth analysis of mAb attributes, is bioactivities due to the increasing demands 4:30 Breakout Discussions: Cell Therapy CMC, employed during characterization. Once attributes Analytical Challenges For Low for processing and formulating antibodies at have been identified and characterized, fast MS-only Quality & Analytics Concentration/Low Dose Drug Products high concentration. I will discuss our work methods capable of monitoring these attributes Cell Therapy Bioproduction, on developing novel methods for identifying, can be more suitable. Here, application of a rapid Dominick DeGrazio, Associate Scientist, Drug Operations & Logistics engineering and characterizing antibodies peptide map method used to monitor relative Product Development, Janssen R&D with reduced propensity to self-associate and amounts of previously characterized mAb attributes • Are there potential solutions to the analytical Gene Therapy Bioproduction increased developability (high solubility, low is discussed. obstacles being seen often with increasingly viscosity, low non-specific interactions). potent molecules? Manufacturing & CMC • For low dose IV or subQ administration, how Strategies for Biologics can pharmacy manual requirements for certain SPONSORS & EXHIBITORS characterization attributes be fulfilled if there is no practical analytical solution available? HOTEL & TRAVEL • What are new, upcoming technologies that could REGISTRATION INFORMATION
BioprocessingSummit.com ◄ 39 ► COVER Formulation & Stability EVENT AT A GLANCE 4th Annual PLENARY KEYNOTE SESSION SHORT COURSES Rapid Methods to Assess Quality & Stability of Biologics TRAINING SEMINARS Improving Prediction and Screening UPSTREAM PROCESSING Optimizing Cell Culture Technology Bioproduction: Scale, Bioreactors provide analytical alternatives, but are currently 8:30 Dimers and Aggregates – Criticality 9:45 Coffee Break in the Exhibit Hall with not commercially viable options yet? & Disposables and Specifications - Are We Barking up the Poster Viewing • What is necessary to develop these Wrong Tree? Optimizing Cell Line Development 10:30 Effect of Particle Density on the technologies as quickly as possible so that they Volker Schnaible, Ph.D., Senior Principal Scientist, Sizing and Counting of Subvisible Protein DOWNSTREAM PROCESSING may be implemented as a qualified, routine Pharma Technical Development Europe (Biologics) analytical tool? Analytics, F. Hoffmann-La Roche AG Aggregates by Orthogonal Methods Continuous Processing in Richard Cavicchi, Ph.D., Physicist, Bioprocess 5:30 Grand Opening Reception in the Theoretical concerns about the role of aggregates/ Biopharm Manufacturing particles as a contributing factor to immunogenicity Measurements Group, National Institute of Advances in Purification Exhibit Hall with Poster Viewing events (e.g. ADA) and the lack of a solid database Standards and Technology Technologies 7:00 End of Day translate into specific requirements from health Measurements of protein aggregates using different authorities. We performed extensive biophysical techniques often result in discrepancies arising Virus & Pathogen Clearance characterization of different dimer species of from the differences in measured properties, e.g. & Safety in Biologics TUESDAY, AUGUST 16 monoclonal antibodies. The immunogenicity flow imaging: area, electrical sensing zone: volume, of these species was evaluated in transgenic resonance mass: mass, etc. A key parameter that ANALYTICAL & QUALITY 7:30 am Registration Opens and Morning mice that are tolerant against human IgG1 but relates the results reported by orthogonal methods Host Cell Proteins: Detection, Coffee immunocompetent. The outcome of these studies is the particle density. We attempt to estimate this Analysis & Removal is presented. parameter by performing two measurements on individual aggregates, and will discuss how this SUBVISIBLE PARTICLES, AGGREGATES Early Analytical Development for 9:00 Particulate Characterization for result can be used to relate measurements by Biotherapeutics & STABILITY Biotherapeutics – Overcoming Technique orthogonal methods. Process Characterization, Challenges and New Learnings 7:55 Chairperson’s Remarks 11:00 Detection and Quantitation of Qualification & Control Ankit Patel, Ph.D., Scientist, Genentech Richard Cavicchi, Ph.D., Physicist, Bioprocess Residuals in Vaccine Components Using Measurements Group, National Institute of In this presentation, we will discuss the inherent FORMULATION & STABILITY NMR Standards and Technology instability of a conjugate type vaccine, various Rapid Methods to Assess reasons for such instability and appropriate Marina Kirkitadze, Ph.D., MBA, Deputy Director, Quality & Stability of Biologics 8:00 Current Technologies for Subvisible mitigation strategy that can be employed. Analytical R&D Biochemistry, Sanofi Pasteur The focus of the presentation is the NMR method Overcoming Formulation Particle Analysis for Biologics 9:30 Selected Poster Presentation: Forced development to determine the limit of quantitation Challenges Andrea Hawe, Ph.D., CSO, Coriolis Pharma An overview on analytical methods for sub- Degradation Study of Biopharmaceutical (LOQ) and limit of detection (LOD) of the process- High-Concentration micrometer and micrometer particles in Antibodies Using Electrochemistry related residuals in the vaccine product candidates. Protein Formulations biopharmaceutical products will be given, with Beijing Huang, Post Doc Researcher, Bioanalytical The results demonstrated that NMR method Science Group, National Institute of Standards and can successfully identify and quantify a residual CELL & GENE THERAPY PRODUCTION respect to measurement principle, capabilities for sizing, quantification and identification, Technology (NIST) in protein component of vaccine and in viral vaccine samples. Cell Therapy CMC, typical applications, and limitations. Methods Forced degradation, the intentional application of Quality & Analytics covered include: field flow fractionation, analytical stress conditions to artificially induce changes in 11:30 Applied Analytical Techniques to Cell Therapy Bioproduction, ultracentrifugation, flow cytometry, laser diffraction, product attribute, is a critical step in the evaluation dynamic light scattering, nanoparticle tracking Monitor IsoAsp in Biologics Formulation Operations & Logistics of pharmaceutical antibodies. Information about analysis, resonant mass measurement, electrical changes that may occur to the antibody after Development Gene Therapy Bioproduction sensing zone analysis, light obscuration, flow exposure to stress conditions can be used to design Dominick DeGrazio, Associate Scientist, Drug imaging and Raman microscopy. production, processing and formulation. In this work Product Development, Janssen R&D Manufacturing & CMC we demonstrate the use of electrochemistry for The development of a suitable biologic formulation Strategies for Biologics controlled and quantitative forced degradation of occurs often before analytical methods are validated. NISTmAb IgG1κ reference material via oxidation. Certain chemical modifications are critical to monitor SPONSORS & EXHIBITORS during the development process as they may cause HOTEL & TRAVEL REGISTRATION INFORMATION
BioprocessingSummit.com ◄ 40 ► COVER Formulation & Stability EVENT AT A GLANCE 4th Annual PLENARY KEYNOTE SESSION SHORT COURSES Rapid Methods to Assess Quality & Stability of Biologics TRAINING SEMINARS Improving Prediction and Screening UPSTREAM PROCESSING Optimizing Cell Culture Technology protein instability and reduce biologic efficacy. Scientist, Biologics Development, Gilead Sciences 3:30 Refreshment Break in the Exhibit Hall Aspartic acid isomerization is one such modification, Bioproduction: Scale, Bioreactors with Poster Viewing but is arguably the most difficult to detect. Analytical 2:00 Automated and Rapid Methods to & Disposables tools to track IsoAsp are discussed that can aid in Assess Quality & Stability of Biologics: 4:15 High Throughput Biophysical and Optimizing Cell Line Development making formulation decisions before the availability Recent Developments and the Keys to Biochemical Stability Screening for Early of qualified methods. Successful Implementation in Formulation Stage Antibody Discovery DOWNSTREAM PROCESSING Sponsored By and Bioprocess Development 12:00 pm Prometheus NT.48: Yingda Xu, Ph.D., Director, Protein Analytics, Adimab Continuous Processing in Danny Chou, Ph.D., President and Founder, Biologics Stability Screening Problems in the development of antibodies Biopharm Manufacturing Compassion BioSolution; Former Senior Research can often be traced back to their intrinsic poor has Never Been Faster, Easier Advances in Purification Scientist, Biologics Development, Gilead Sciences biophysical and biochemical stabilities. High & More Fun Technologies The goal of this presentation is to describe new throughput screening assays are developed or Dennis Breitspecher, Ph.D., Head, Research developments in the field of analytical technologies adapted to fit in the scope of early discovery stage Virus & Pathogen Clearance & Development, Biochemistry Research & for protein aggregate testing and characterization. to filter out candidates with poor properties. & Safety in Biologics Development, NanoTemper Technologies, GmbH Particular focus will be on techniques that are Prometheus NT.48 allows for rapid and precise automated, high throughput, and capable of 4:45 Panel Discussion: Challenges in ANALYTICAL & QUALITY high-throughput stability screenings, while providing detecting subvisible particulates. Methods that Adopting New Methods in QC Labs Host Cell Proteins: Detection, best-in-class high resolution thermal unfolding detect changes in other quality attributes will also Moderator: Analysis & Removal data for biologics, independent of buffer or protein be discussed. Danny Chou, Ph.D., President and Founder, concentration. It allows for an exact detection of Early Analytical Development for Compassion BioSolution; Former Senior Research aggregation onset temperatures and aggregation 2:30 Analytical Methods in Support of Scientist, Biologics Development, Gilead Sciences Biotherapeutics behavior to find the conditions in which the protein High Throughput Conjugation Process Panelists: is most stable. Development for ADCs Process Characterization, Michael Fleming, MS, Scientist, Analytical and Michael Fleming, MS, Scientist, Analytical and Qualification & Control 12:15 Meet Our UNcle: Sponsored By Pharmaceutical Sciences, ImmunoGen, Inc. Pharmaceutical Sciences, ImmunoGen, Inc. FORMULATION & STABILITY the All-In-One Biologics Pilsoo Kang, Ph.D., Scientist I, Analytical Science Assays that are suitable of providing rapid results and Technology, Sanofi Rapid Methods to Assess Stability Platform about important ADC related attributes (e.g. size Yingda Xu, Ph.D., Director, Protein Analytics, Adimab Quality & Stability of Biologics Joe Barco, Director, Product Marketing, Marketing, and charge variants, drug loading, residual free small Unchained Labs molecules) will be discussed in this presentation. 5:15 End of Conference Overcoming Formulation Characterizing biologic stability is a wrestling match Challenges with disjointed data from sub-optimized tools. Not 3:00 Development of High-Throughput 5:15 Registration for Dinner Short Course High-Concentration anymore. UNcle combines fluorescence, SLS, Quantitative UPLC-High Resolution Mass Protein Formulations and DLS reads in one instrument. This allows you Spectrometry Multi-Attribute Methodology 6:00 – 8:30 Recommended Dinner Short to quickly and efficiently characterize all the size, for Biologics Course* CELL & GENE THERAPY PRODUCTION aggregation, thermal ramp, isothermal and viscosity Pilsoo Kang, Ph.D., Scientist I, Analytical Science Protein Aggregation: Mechanism, Characterization information you want. Cell Therapy CMC, and Technology, Sanofi and Consequences Quality & Analytics Understanding product quality attributes is * Separate registration required, see page 5 for details. 12:30 Luncheon Presentation (Sponsorship Cell Therapy Bioproduction, crucial in Quality by Design approach drug Opportunity Available) or Lunch on development. To accomplish this, appropriate Operations & Logistics Your Own analytical methodologies are required. Generally, Gene Therapy Bioproduction the number of analytical methods used in protein 1:15 Dessert Refreshment Break in the characterization is large. To increase efficiency, Manufacturing & CMC Exhibit Hall with Poster Viewing we developed a quantitative UPLC-high resolution Strategies for Biologics mass spectrometry-based method which permits monitoring multiple quality attributes of biologics SPONSORS & EXHIBITORS HIGH-THROUGHPUT & HIGH RESOLUTION SCREENING products. This multi-attribute methodology can be HOTEL & TRAVEL applied to other products as a platform approach. REGISTRATION INFORMATION 1:55 Chairperson’s Remarks Danny Chou, Ph.D., President and Founder, Compassion BioSolution; Former Senior Research BioprocessingSummit.com ◄ 41 ► COVER Formulation & Stability EVENT AT A GLANCE 4th Annual PLENARY KEYNOTE SESSION SHORT COURSES Overcoming Formulation Challenges for TRAINING SEMINARS UPSTREAM PROCESSING Biopharmaceutical Development Optimizing Cell Culture Technology Formulation and Process Optimization, Analytics, Device Integration and New Biologics Bioproduction: Scale, Bioreactors Sponsored By & Disposables WEDNESDAY, AUGUST 17 9:30 Inherent Instability of a Conjugate Type 11:45 Automated Buffer Vaccine- Cause and Mitigation Strategy Exchange and Concentration Optimizing Cell Line Development 7:00 am Registration Opens and Morning Sumit Goswami, Ph.D., Principal Scientist, of Biopharmaceuticals DOWNSTREAM PROCESSING Coffee Pharmaceutical R&D, Biotherapeutics Taegen Clary, Vice President, Marketing, Pharmaceutical Sciences, Pfizer, Inc. Unchained Labs Continuous Processing in FORMULATION DEVELOPMENT OF In this presentation, we will discuss the inherent Protein formulation preparation requires either Biopharm Manufacturing NOVEL BIOLOGICS instability of a conjugate type vaccine, various dilution or buffer exchange of a protein solution into Advances in Purification reasons for such instability and appropriate multiple formulation buffers. Traditional approaches Technologies 8:05 Chairperson’s Opening Remarks mitigation strategy that can be employed. for buffer exchange include dialysis, desalting columns, and centrifugal UF/DF devices. While Virus & Pathogen Clearance Christian Schöneich, Ph.D., Takeru Higuchi Distinguished Professor and Chair, Pharmaceutical 10:00 Coffee Break in the Exhibit Hall with these are all relatively simple and easy they are & Safety in Biologics Chemistry, University of Kansas Poster Viewing also manual and specifically, dialysis and desalting columns, do not allow for protein concentration. ANALYTICAL & QUALITY KEYNOTE PRESENTATIONS: CRITICAL CONSIDERATIONS FOR Unchained Labs offers multiple systems for Host Cell Proteins: Detection, FORMULATION OPTIMIZATION: NEW automated buffer exchange and concentration Analysis & Removal 8:15 Considerations about Development of biopharmaceuticals. In this talk we describe EXCIPIENTS, SURFACTANTS & MORE these systems and their application to protein Early Analytical Development for of Stable Liquid Formulations for formulation preparation. Biotherapeutics Maytansinoid ADCs 10:45 Effects of Arginine Counter Ions in Alex Lazar, Ph.D., Head of Analytical and Process Characterization, Solubility, Protein-Protein Interaction, and 12:15 Luncheon Presentation (Sponsorship Pharmaceutical Sciences, ImmunoGen, Inc. Qualification & Control Stability of a Monoclonal Antibody Opportunity Available) or Lunch on Your Own The attachment of the small molecules in ADCs Haripada Maity, Ph.D., Research Advisor, FORMULATION & STABILITY changes the physicochemical properties of the Formulation Development, CMC Development, Eli 1:00 Session Break proteins. In order to stabilize the ADCs, we need Lilly and Company Rapid Methods to Assess to study their behavior under different conditions The most commonly used salt of arginine in protein CRITICAL CONSIDERATIONS FOR Quality & Stability of Biologics in order to design appropriate formulations. formulation development is arginine hydrochloride. FORMULATION OPTIMIZATION: NEW Overcoming Formulation Although anions are considered to be more effective Challenges 9:00 Glycoform Diversity and Antibody- EXCIPIENTS, SURFACTANTS & MORE Drug Conjugation Control the Chemical compared to cations, a limited number of studies High-Concentration have been performed at understanding the effects 1:45 Chairperson’s Remarks Stability of Antibodies of counter ions of arginine on various solution Protein Formulations Christian Schöneich, Ph.D., Takeru Higuchi Haripada Maity, Ph.D., Research Advisor, properties of proteins. This presentation will discuss Formulation Development, CMC Development, Eli CELL & GENE THERAPY PRODUCTION Distinguished Professor and Chair, the effects of different counter ions and will shed Pharmaceutical Chemistry, University of Kansas Lilly and Company Cell Therapy CMC, some light on understanding the mechanism of Antibodies are target for a large variety of arginine-X (X refers to counter ion) interaction Quality & Analytics 1:50 Polysorbate 20 Degradation and Free chemical degradation reactions during processing with proteins. Fatty Acid Release in a Sulfatase Drug and storage. This presentation will provide Cell Therapy Bioproduction, Product: Identification of Residual Host Cell Operations & Logistics new examples, where forced degradation 11:15 Formulation of High Concentration studies have led to the characterization of novel Monoclonal Antibodies to Minimize Protein as a Potential Root Cause Gene Therapy Bioproduction degradation products generated during storage. Aggregation Caused by Distinctive Protein Nitin Dixit, Ph.D., Scientist, Drug & Combination Experimental evidence for the effect of glycoform Product Development, Shire Isoforms Manufacturing & CMC diversity and antibody-drug conjugation on This study investigated the root cause behind an Ionela Iliescu, M.Sc., Scientist I, Technical Strategies for Biologics the chemical degradation of antibodies will observed free fatty acid particle formation and Development, Biogen SPONSORS & EXHIBITORS be presented. Polysorbate 20 loss in a sulfatase drug product upon long term storage. The current work highlights the HOTEL & TRAVEL importance of early detection of potential impurities REGISTRATION INFORMATION in the protein drug substance that may contribute
BioprocessingSummit.com ◄ 42 ► COVER Formulation & Stability EVENT AT A GLANCE 4th Annual PLENARY KEYNOTE SESSION SHORT COURSES Overcoming Formulation Challenges for TRAINING SEMINARS UPSTREAM PROCESSING Biopharmaceutical Development Optimizing Cell Culture Technology Formulation and Process Optimization, Analytics, Device Integration and New Biologics Bioproduction: Scale, Bioreactors & Disposables to polysorbate degradation to make a judicious 3:50 Refreshment Break in the Exhibit Hall Combination Product Development, Shire selection of the surfactant and its optimized with Poster Viewing Forced degradation is widely used through the Optimizing Cell Line Development concentration for the final drug product. life cycle of biological product development. 4:45 Plenary Keynote Session (see page 3 DOWNSTREAM PROCESSING Implementation of properly designed forced 2:20 Alternative Surfactants: A Search for for details) degradation studies can improve the efficiency of Continuous Processing in Substitutes to Polysorbates product development. This presentation focuses on Biopharm Manufacturing Kishore Ravuri, Ph.D., Principal Scientist, 6:00 Networking Reception in the Exhibit application of QbD strategy to assist the design and Advances in Purification Formulation Development, Europe Biologics, F. Hall with Poster Viewing implementation of forced degradation studies for Hoffmann-La Roche efficient product development. A case study will be Technologies 7:00 End of Day Surfactants are often indispensable components of provided to demonstrate the QbD strategy based Virus & Pathogen Clearance biologic, parenteral formulations. Polysorbates have study design, implementation time, and application & Safety in Biologics always been the best choice till date. Recent studies THURSDAY, AUGUST 18 of data package. have pointed towards certain disadvantages which ANALYTICAL & QUALITY Polysorbates might pose if not carefully monitored. 8:00 am Registration Opens and Morning 9:30 Analytical Challenges and Control Host Cell Proteins: Detection, This could range from surfactant degradation to Coffee Strategies for High Concentration Analysis & Removal peroxide induced Drug Product oxidation. With the Formulations and Associated Delivery increasing number of novel protein formats entering Devices Early Analytical Development for ACCELERATING BIOLOGICS DRUG the research pipeline, it is worthwhile to look for DEVELOPMENT Abbas Razvi, Ph.D., Formulation Development, Biotherapeutics potential alternatives to polysorbates which can Lonza AG Process Characterization, overcome challenges which polysorbates tend to 8:25 Chairperson’s Remarks Biological drug products are complex systems pose. The current talk focuses on our evaluation of Qualification & Control Mark L. Brader, Ph.D., Independent Consultant - comprised of several components in addition to the alternatives to polysorbates. API, including excipients, stabilizers, a container FORMULATION & STABILITY Protein Drug Product Formulation and Biophysical Characterization and container closure system. High concentration Rapid Methods to Assess 2:50 i-Raman Portable Spectrometer formulations pose challenges related to API stability Quality & Stability of Biologics to Identify Raw Materials and Active 8:30 Bringing Back the Good Stuff: X-Ray and stabilizer and excipient degradation. Successful Components of Novel Vaccines high concentration formulation and delivery Overcoming Formulation Crystallography to Accelerate Biologics Marina Kirkitadze, Ph.D., MBA, Deputy Director, Drug Development systems require an effective control strategy that Challenges Analytical R&D Biochemistry, Sanofi Pasteur addresses all aspects of this complex drug product. Mark L. Brader, Ph.D., Independent Consultant - This presentation will review current knowledge High-Concentration The topic of this presentation is Raman Protein Drug Product Formulation and Biophysical on high concentration formulations and associated Protein Formulations Spectroscopy method development to identify Characterization biological raw materials for a novel recombinant analytical challenges. X-ray crystallography enables atomic-level insight CELL & GENE THERAPY PRODUCTION adjuvanted vaccine, and active components into protein structure, function, and mechanistic and excipients of novel viral vaccine during the 10:00 Coffee Break in the Exhibit Hall with Cell Therapy CMC, pathways in biology. It plays a prominent role in formulation. The advantages of using i-Raman Poster Viewing Quality & Analytics structure-based lead discovery yet is largely ignored portable spectrometer will be discussed. Cell Therapy Bioproduction, as a characterization tool during development. 10:45 Selected Poster Presentation: Operations & Logistics 3:20 Storage and Delivery of This presentation will examine some common Implementation of USP Antibody Standard myths surrounding protein crystallization and Biopharmaceuticals: Identifying Risk in for System Suitability in Capillary Gene Therapy Bioproduction x-ray crystallography, and explore how recent Electrophoresis Sodium Dodecyl Sulfate Early Development Stages advancements allow this technique to be applied in Manufacturing & CMC Diane Paskiet, Senior Director of Global Scientific new and creative ways to aid the development of (CE-SDS) for Release and Stability Methods Strategies for Biologics Affairs, West Pharmaceutical complex therapeutic biomacromolecules. Abbie L. Esterman, Ph.D., Senior Scientist, Molecular and Analytical Development, Biologics SPONSORS & EXHIBITORS 9:00 QbD Based Forced Degradation -- The Manufacturing & Process Development, Bristol- HOTEL & TRAVEL Key of Efficient Drug Product Development Myers Squibb Yu Tang, Ph.D., Principal Scientist, Drug & REGISTRATION INFORMATION
BioprocessingSummit.com ◄ 43 ► COVER Formulation & Stability EVENT AT A GLANCE 4th Annual PLENARY KEYNOTE SESSION SHORT COURSES Overcoming Formulation Challenges for TRAINING SEMINARS UPSTREAM PROCESSING Biopharmaceutical Development Optimizing Cell Culture Technology Formulation and Process Optimization, Analytics, Device Integration and New Biologics Bioproduction: Scale, Bioreactors & Disposables 11:00 Selected Poster Presentation 11:45 Panel Discussion: Special Exploring Inline Raman Method Considerations for Biophysical Analysis in Optimizing Cell Line Development Development through Better Formulation Development DOWNSTREAM PROCESSING Understanding of Spectral Interferences Moderator: Continuous Processing in Dimuthu Jayawickrama, Ph.D., Sr. Research Mark L. Brader, Ph.D., Independent Consultant - Biopharm Manufacturing Investigator II, Drug Science Product & Technology, Protein Drug Product Formulation and Biophysical Bristol Myers Squibb Characterization Advances in Purification Raman spectroscopy is increasingly used as a Panelists: Technologies real-time spectroscopic tool in mammalian cell Mark L. Brader, Ph.D., Independent Consultant - Virus & Pathogen Clearance culture bioreactors for monitoring of nutrients Protein Drug Product Formulation and Biophysical and metabolites. Generally the Raman calibration & Safety in Biologics Characterization methods for bioreactors are developed with spectral Yu Tang, Ph.D., Principal Scientist, Drug & ANALYTICAL & QUALITY data generated with multiple batches manufactured Combination Product Development, Shire under similar conditions. This approach is Joanna MacKay, Ph.D., Scientist, Drug Product Host Cell Proteins: Detection, practical at manufacturing where process is well Development, Janssen R&D Analysis & Removal understood and controlled. However this approach of developing methods may not be suitable Christopher Mensch, Scientist, Vaccine Drug Early Analytical Development for Product Development, Merck Biotherapeutics during process development where materials and process conditions can vary greatly. Therefore, 12:15 Lunch Available for Purchase in the Process Characterization, an alternative approach of method development Exhibit Hall Qualification & Control has been explored. The objective is to develop robust methods independent of or less sensitive FORMULATION & STABILITY 1:15 Dessert Refreshment Break in the to cell type, cell population, and cell debris and Exhibit Hall with Poster Viewing Rapid Methods to Assess fluorescence background. This work describes a Quality & Stability of Biologics series of experiments designed to isolate and study 1:55 End of Conference various interferences as described above. Potentially Overcoming Formulation remedies to eliminate these interferences Challenges are presented. High-Concentration Protein Formulations 11:15 Overcoming Freeze-Thaw Agglomeration of a Meningococcal Vaccine CELL & GENE THERAPY PRODUCTION Christopher Mensch, Scientist, Vaccine Drug Cell Therapy CMC, Product Development, Merck Quality & Analytics The purpose of this work was to investigate the susceptibility of an aluminum adjuvant and an Cell Therapy Bioproduction, aluminum-adjuvanted native outer membrane Operations & Logistics vesicle (nOMV) vaccine formulation to freeze/ Gene Therapy Bioproduction thaw–induced agglomeration using static light scattering and micro-flow Imaging analysis; and to Manufacturing & CMC evaluate the use of propylene glycol as a vaccine Strategies for Biologics formulation excipient by which freeze/thaw–induced agglomeration of a nOMV vaccine formulation could SPONSORS & EXHIBITORS be mitigated. Our results indicate that including 7% HOTEL & TRAVEL v/v propylene glycol in a nOMV containing aluminum adjuvanted vaccine formulation, mitigates freeze/ REGISTRATION INFORMATION thaw–induced agglomeration.
BioprocessingSummit.com ◄ 44 ► COVER Formulation & Stability EVENT AT A GLANCE 5th Annual PLENARY KEYNOTE SESSION SHORT COURSES High-Concentration Protein Formulations TRAINING SEMINARS Formulation, Manufacturing, Analytics, High Viscosity, Aggregation, Devices and Delivery UPSTREAM PROCESSING THURSDAY, AUGUST 18 5:15 End of Day KEYNOTE PRESENTATION: Optimizing Cell Culture Technology 11:30 am Registration Opens 5:15 Registration for Dinner Short Course Bioproduction: Scale, Bioreactors 2:45 Applications of AU-FDS to High- & Disposables 12:15 pm Lunch Available for Purchase in Concentration Antibody Interactions Thomas Laue, Ph.D., Professor, Biochemistry FRIDAY, AUGUST 19 the Exhibit Hall Optimizing Cell Line Development and Molecular Biology; Director, Biomolecular 8:00 am Registration Opens and Morning DOWNSTREAM PROCESSING 1:15 Dessert Refreshment Break in the Interaction Technologies Center (BITC), University of New Hampshire Coffee Continuous Processing in Exhibit Hall with Poster Viewing Will discuss two applications of AU-FDS. First, Biopharm Manufacturing PROTEIN-PROTEIN INTERACTIONS, high-concentration antibody solutions may exhibit PROCESS CHALLENGES Advances in Purification excessive viscosity due to weak interactions VISCOSITY & AGGREGATION 8:25 Chairperson’s Remarks Technologies between proteins. A case will be built that Mark Yang, Ph.D., Director, Fill Finish Development, 1:55 Chairperson’s Opening Remarks they are a consequence of a balance between Virus & Pathogen Clearance attractive electrostatic energies and repulsive Sanofi Genzyme & Safety in Biologics Atul Saluja, Ph.D., Senior Research Investigator II, desolvation energies. Second, how does a high- 8:30 Detection and Management of Protein Drug Product Science & Technology, Bristol-Myers concentration protein background influence an ANALYTICAL & QUALITY Squibb IgG-antigen interaction? Here, both the IgG and Aggregates during Downstream Processing Host Cell Proteins: Detection, the antigen are multi-valent, which leads to very Danny Chou, Ph.D., President and Founder, Analysis & Removal KEYNOTE PRESENTATION: large networks being formed in dilute solution. Compassion BioSolution; Former Senior Research Are these large structures observed in serum? Scientist, Biologics Development, Gilead Sciences Early Analytical Development for 2:00 Correlating Protein-Protein It is known that protein aggregation is a significant Biotherapeutics Interactions and Aggregation Rates 3:15 Use of Viscosity Surrogate Solutions bottleneck in the development of efficient and cost- over Broad Concentration Range: effective biologics purification process. The goal of Process Characterization, to Identify Equipment Operating Qualification & Control Misconceptions, Challenges and this presentation is to use a case study to illustrate Promising Approaches Conditions in Fill-Finish Process for High how one may detect the “seeds” of protein FORMULATION & STABILITY Atul Saluja, Ph.D., Senior Research Investigator Concentration Biologic Drug Product aggregate that occur early in the process in order to Rapid Methods to Assess II, Drug Product Science & Technology, Bristol- Zach Zhu, Ph.D., Scientist II, Pharmaceutical reduce the formation of larger particulate in the final Operations & Technology, Biogen Quality & Stability of Biologics Myers Squibb drug product and reduce processing cost. Protein–protein interactions (PPI) and rates of A platform approach was applied to the 9:00 Case Study: Formulation pH Shift Overcoming Formulation aggregation (kagg) were quantified systematically development of several fill-finish process unit Challenges for a monoclonal antibody (MAb) across a operations for a highly concentrated biologic drug Caused by Protein Concentration Increase High-Concentration broad range of concentration in presence and product. The approach utilized viscosity surrogate Mark Yang, Ph.D., Director, Fill Finish Development, solutions to identify operational parameters for Protein Formulations absence of excipients. Hypotheses based on Sanofi Genzyme thermodynamic activity and fluctuation theory a scaleable single-use system (SUS), which is In this new case study, it has been observed CELL & GENE THERAPY PRODUCTION were inconsistent with experimental kagg and applied for mixing, pooling, and filtration steps in that the formulation pH decreases as its protein the manufacturing operation. Accordingly, at-scale scattering data. However, arguments based on concentration increases. This pH shift becomes Cell Therapy CMC, equipment operating conditions were successfully Quality & Analytics surface contact probabilities were reasonably more pronounced in the high concentration successful in providing a semi-quantitative identified, leading to a significant reduction in formulations or in the diafiltration step where Cell Therapy Bioproduction, correlation. Challenges and opportunities for such characterization of the commercial-scale process protein gets concentrated. The underlining Operations & Logistics an approach are further discussed. using active product. A GMP production run of mechanisms responsible for this pH shift, along with active drug product further confirmed the suitability mitigation strategies, will be discussed. Gene Therapy Bioproduction of process parameters identified from the surrogate solutions. Manufacturing & CMC 9:30 Impact of Charge Isoforms on Stability 3:45 Sponsored Presentation (Opportunity Strategies for Biologics of Antibody Formulations Manufactured Available) Using High Titer Bioprocess SPONSORS & EXHIBITORS Jason Fernandez, M.Sc., Scientist, Protein HOTEL & TRAVEL 4:00 Refreshment Break Pharmaceutical Development, Biogen This presentation will cover how molecular charge REGISTRATION INFORMATION 4:15 Breakout Discussions isoforms from upstream drug substance process influence protein stability in the downstream drug BioprocessingSummit.com ◄ 45 ► COVER 5th Annual Formulation & Stability EVENT AT A GLANCE PLENARY KEYNOTE SESSION High-Concentration Protein Formulations SHORT COURSES Formulation, Manufacturing, Analytics, High Viscosity, Aggregation, Devices and Delivery TRAINING SEMINARS UPSTREAM PROCESSING product. Particular post-translational modifications low-volume devices. challenging formulations. and chemical degradation will be discussed that lead Optimizing Cell Culture Technology to charge isoform impacting protein aggregation. 11:30 Building a Better Bridge: 2:00 Product Differentiation through Bioproduction: Scale, Bioreactors Further, isoform fractionation approaches and novel Biophysical Tools to Better Understand Formulation and Device & Disposables stability study designs will be presented that help the Complexities of High Concentration Jan Jezek, Ph.D., CSO, Research & Development, elucidate the impact of isoform species in high titer Biotherapeutics Arecor, Ltd. Optimizing Cell Line Development and high concentration biopharmaceuticals. Mike Marlow, Ph.D., Senior Staff Scientist, Protein In the increasingly competitive biopharmaceutical DOWNSTREAM PROCESSING Biochemistry, Regeneron Pharmaceuticals, Inc. market, convenience of use has become an 10:00 Networking Coffee Break essential feature for a product to succeed. The thermodynamic interactions that govern a Continuous Processing in Convenience can be achieved by smart devices Biopharm Manufacturing variety of protein therapeutic and solution properties FORMULATION & ANALYTICAL are distinctly protein concentration dependent. as well as formulations that enable high protein Advances in Purification STRATEGIES FOR HIGH- Manufacturing and dosing of therapeutic monoclonal concentration or use of the product outside the cold chain. This talk will focus on unique formulation Technologies CONCENTRATION PROTEIN antibodies frequently calls for high protein concentration solutions and the resulting non-ideality strategies for stable concentrated products as well Virus & Pathogen Clearance FORMULATIONS complicates reliable estimation of critical properties as related device choices that enable development & Safety in Biologics from measurements under dilute conditions. We of competitive biopharmaceuticals. 10:30 Co-Formulation Development of ANALYTICAL & QUALITY illustrate the utility of different biophysical tools Monoclonal Antibodies and Recombinant in bridging the dilute - high concentration gap by 2:30 Mitigation of High Concentration Host Cell Proteins: Detection, Human Hyaluronidase (rHuPH20) – A Case characterizing two antibodies that exhibit contrasting Formulation Issues of a Human IgG1 Analysis & Removal Study concentration-dependent behavior. Monoclonal Antibody via Rational, Early Analytical Development for Claudia Mueller, Ph.D., Senior Scientist, Group Structure- Guided Protein Engineering 12:00 pm Sponsored Presentations Biotherapeutics Leader, Late-Stage Pharmaceutical and Process Reza Esfandiary, Ph.D., Scientist II, Department of Development, F. Hoffmann-La Roche Ltd. (Opportunities Available) Formulation Sciences, MedImmune Process Characterization, Subcutaneous application faces limitations High concentrations can add significant challenges 12:30 Luncheon Presentation (Sponsorship Qualification & Control with regards to the drug volume that can be during development of mAb therapeutics. Although administered. To enable delivery of the necessary Opportunity Available) or Lunch on Your Own a global mechanistic understanding might be FORMULATION & STABILITY doses, increase in the concentration of the drug 1:15 Session Break sufficient to develop robust formulation controls, Rapid Methods to Assess and/or temporary enlargement of the interstitial mitigation approaches via protein engineering Quality & Stability of Biologics space at the injection site, e.g. by using rHuPH20, DELIVERY & DEVICES FOR HIGH- requires knowledge of the molecular hotspots. Overcoming Formulation are potential strategies to face this challenge. Here, we have integrated experimental and in silico This presentation highlights the drug product CONCENTRATION / HIGH-VOLUME methods to systematically identify the molecular Challenges development of co-formulations consisting of highly FORMULATIONS hotspots responsible for high concentration High-Concentration concentrated monoclonal antibodies and rHuPH20. issues of a model mAb and have engineered Protein Formulations The talk will focus on formulation and process 1:25 Chairperson’s Remarks variants with improved high concentration development challenges arising from combination of Jan Jezek, Ph.D., CSO, Research & Development, developability properties. CELL & GENE THERAPY PRODUCTION two very different proteins in order to maintain both Arecor Ltd. Cell Therapy CMC, stable and active within one formulation. 3:00 CMC Strategies on Scaling Up and Quality & Analytics 1:30 Strategies for Overcoming Long Down for High-Concentration Protein 11:00 Formulation Strategies for High- Reconstitution Times of Lyophilized Highly Formulations Cell Therapy Bioproduction, Concentration, Low-Volume Injectables Concentrated Protein Formulations Jamie Tsung, Ph.D., Principal Scientist, Momenta Operations & Logistics Charles Wescott, Ph.D., Vice President, Research Robin Bogner, Ph.D., Associate Professor, Pharmaceuticals, Inc. Gene Therapy Bioproduction and Development, Protein Formulation, Arsia Pharmaceutical Sciences, University of Connecticut Highly concentrated therapeutic proteins are prone Therapeutics High protein concentration is often accompanied to be viscous and aggregate posing developmental Manufacturing & CMC At concentrations exceeding 100 mg/mL, many by unacceptably long reconstitution times of 40-60 and CMC challenges in purification, formulation, Strategies for Biologics biologics are highly viscous, and can suffer reduced minutes. Formulation and lyophilization cycles analytical development, manufacturing, product colloidal stability. Formulation designs targeting stability, syringeability and injectability. This SPONSORS & EXHIBITORS have been found to influence the time to achieve the protein-protein interactions mediating these complete reconstitution. In addition, there are talk provides a review of current strategies and HOTEL & TRAVEL problems can produce highly-concentrated drug an increasing battery of methods to assess the technologies used in product development to products with good stability, syringeability, and potential for long reconstitution times. Several overcome these CMC challenges and minimize the REGISTRATION INFORMATION injectability characteristics. These advanced strategies, some of which may be counterintuitive, impact on product quality. high-concentration formulations allow biologics can be used to reduce the reconstitution times of BioprocessingSummit.com that would otherwise be dosed by i.v. infusion to 3:30 Close of Conference be administered by s.c. injection, or delivered by ◄ 46 ► COVER EVENT AT A GLANCE PLENARY KEYNOTE SESSION STREAM #5 SHORT COURSES TRAINING SEMINARS UPSTREAM PROCESSING Cell & Gene Therapy Production Optimizing Cell Culture Technology Bioproduction: Scale, Bioreactors & Disposables This week-long Cell and Gene Therapy Production stream focuses on the analysis, process development, scale-up and Optimizing Cell Line Development manufacture of cell and gene-based products, with particular attention on CAR-T and TCR-based therapies. First stop is Cell DOWNSTREAM PROCESSING Continuous Processing in Therapy CMC, looking at potency assay development, raw materials, comparability, target product profiles and quality control, Biopharm Manufacturing followed by two days on cell therapy bioproduction – from scale-up to automation and closed systems; operations to logistics. Advances in Purification Technologies The final part of the week focuses on gene therapy and the challenges in vector design, development and manufacture for AAV Virus & Pathogen Clearance and lentiviruses, with supplementary sessions on analytics and CMC. & Safety in Biologics ANALYTICAL & QUALITY Host Cell Proteins: Detection, Analysis & Removal Early Analytical Development for AUGUST 15-16 Biotherapeutics Process Characterization, Cell Therapy CMC, Quality & Analytics Qualification & Control FORMULATION & STABILITY Rapid Methods to Assess AUGUST 17-18 Quality & Stability of Biologics Overcoming Formulation Cell Therapy Bioproduction, Operations & Logistics Challenges High-Concentration Protein Formulations AUGUST 18-19 CELL & GENE THERAPY PRODUCTION Cell Therapy CMC, Gene Therapy Bioproduction and CMC Quality & Analytics Cell Therapy Bioproduction, Operations & Logistics Gene Therapy Bioproduction Manufacturing & CMC Strategies for Biologics SPONSORS & EXHIBITORS HOTEL & TRAVEL REGISTRATION INFORMATION
BioprocessingSummit.com COVER Cell & Gene Therapy Production EVENT AT A GLANCE Inaugural PLENARY KEYNOTE SESSION SHORT COURSES Cell Therapy CMC, Quality and Analytics TRAINING SEMINARS Understanding Your Product and Process for Late-Stage Success UPSTREAM PROCESSING Optimizing Cell Culture Technology MONDAY, AUGUST 15 2:15 Ensuring Quality of Cellular Starting Gene Therapy Bioproduction: Scale, Bioreactors Material • Global Regulatory Updates and Developments & Disposables 8:00 am Short Course Registration Elizabeth Read, M.D., Senior Vice President, Product • Building a Regulatory Strategy Optimizing Cell Line Development Development, Medeor Therapeutics, Inc. • Common Regulatory Issues 9:00 – 11:30 Recommended Morning For cell therapy products, the starting material DOWNSTREAM PROCESSING Short Course* contains living, functional cells that become an 4:30 Breakout Discussions Continuous Processing in Advanced Process Control Strategies integral part of the active drug substance and 5:30 Grand Opening Reception in the Biopharm Manufacturing in Bioprocessing final product. Quality specifications for the donor, * Separate registration required, see page 5 for details. collection process, and shipping/storage/hold steps Exhibit Hall with Poster Viewing Advances in Purification of starting material should be considered early Technologies 11:30 Main Conference Registration Opens in CMC development. Donor-to-donor variability, 7:00 End of Day Virus & Pathogen Clearance availability of clinically representative starting LATEST TRENDS & RAW MATERIALS materials, and starting material stability create TUESDAY, AUGUST 16 & Safety in Biologics unique development challenges. ANALYTICAL & QUALITY 1:00 pm Chairperson’s Opening Remarks 7:30 am Registration Opens and Morning 2:45 Refreshment Break Christopher Bravery, Ph.D., Consultant Regulatory Coffee Host Cell Proteins: Detection, Scientist, Consulting on Advanced Biologicals Ltd. Analysis & Removal STARTING MATERIALS & POTENCY ASSAYS, QBD & PRODUCT 1:10 KEYNOTE PRESENTATION: Early Analytical Development for INTERNATIONAL REGULATORY QUALITY Biotherapeutics Classification of Cell Therapies and UPDATES Recent Trends Process Characterization, 7:55 Chairperson’s Remarks 3:15 Developing a Clinical Grade Process Qualification & Control Alexey Bersenev, Ph.D., Director, Advanced Cell Bernadette Keane, Ph.D., Consultant, CMC/Quality/ Therapy Lab, Yale University Starting from a Research Method: Practical Regulatory Compliance, Specializing in Gene and FORMULATION & STABILITY Cell therapy is administration of living cells Consideration of Raw Materials for Cell Cell Therapies Rapid Methods to Assess in human with therapeutic purpose. In this Therapy Production 8:00 Overcoming Challenges in Developing Quality & Stability of Biologics presentation I will discuss classification and Benjamin Fryer, Ph.D., Team Leader, Processing/ categories of cell therapies in terms of cell types, Manufacturing, Heart Regeneration Program, Potency Assays for Heterogeneous Cell Overcoming Formulation therapeutic purpose, degree of manipulation and University of Washington School of Medicine Products regulatory pathways. I will highlight differences Challenges In order to generate an approved cell therapy an Therese Choquette, Ph.D., Fellow, TRD Cell & Gene between immunocellular therapies and High-Concentration aseptic and well controlled manufacturing process Therapy, Novartis Pharmaceuticals regenerative medicine and present recent trends must be developed. This begins with proper raw The ideal potency assay reflects the mechanism Protein Formulations in academic and commercial development of material sourcing via secure and trust worthy supply of action (MoA), predicts clinical response, is easy advanced cell therapies. CELL & GENE THERAPY PRODUCTION chains, and material validation via identity and/or to perform and shows low variability. However, for in-process testing. Planning ahead in early stage cell and gene therapy products, often containing Cell Therapy CMC, 1:45 Defining and Qualifying Raw Materials Quality & Analytics development should facilitate development of a heterogenic cell populations, this ideal is rarely for Use in Cell Therapies scalable cold supply chain cell therapy product. achievable. Several challenges exists; including Cell Therapy Bioproduction, Fouad Atouf, Ph.D., Director, United States unknown MoA, bystander activations, unspecific Operations & Logistics Pharmacopeia 3:45 Sponsored Presentation (Opportunity responses, and patient-to-patient variations. This Gene Therapy Bioproduction Qualification of raw materials used in the Available) presentation illustrates some of our experiences of manufacturing of cellular therapies, require the use potency assay development for CART cells. 4:00 Panel Discussion: Navigating Manufacturing & CMC of risk assessment strategies to categorize the International Regulations for Cell and Gene 8:30 Fluorescent Probe-Based Platform for Strategies for Biologics critical components of a manufacturing process. In addition to cell culture supplements, excipients Therapies Mesenchymal Stem Cell Quality Control SPONSORS & EXHIBITORS and other formulation’s components must meet Panelists: Christopher Bravery, Ph.D., Consultant Deepak Raghothaman, PhD., Project Scientist, the required quality to ensure consistency in HOTEL & TRAVEL Regulatory Scientist, Consulting on Advanced Bioprocessing Technology Institute (BTI), A*STAR manufacturing and subsequently the quality and Biologicals Ltd. REGISTRATION INFORMATION safety of finished cell therapy products. Testing Scott Burger, M.D., Consultant, Advanced Cell and strategies for raw materials may be achieved by the BioprocessingSummit.com use of established and validated procedures as well as the availability of reference materials. ◄ 48 ► COVER Cell & Gene Therapy Production EVENT AT A GLANCE Inaugural PLENARY KEYNOTE SESSION SHORT COURSES Cell Therapy CMC, Quality and Analytics TRAINING SEMINARS Understanding Your Product and Process for Late-Stage Success UPSTREAM PROCESSING Optimizing Cell Culture Technology 11:30 Early Process Development of CAR-T 1:15 Dessert Refreshment Break in the Therapies Exhibit Hall with Poster Viewing Bioproduction: Scale, Bioreactors 9:00 The Challenge of Maintaining Product & Disposables Ryan Crisman, PhD., Associate Director, Head Consistency for Autologous Cell Therapies of Late Stage Process Development, Juno RELEASE CRITERIA & Optimizing Cell Line Development Bo Kara, Head, Process Development, Cell Gene Therapeutics Therapy, Platform CMC, GSK COMPARABILITY STUDIES DOWNSTREAM PROCESSING 12:00 pm Ensuring Security Sponsored By 1:55 Chairperson’s Remarks Continuous Processing in 9:30 Sponsored Presentation (Opportunity Within the Cell Therapy Available) Bernadette Keane, Ph.D., Consultant, CMC/Quality/ Biopharm Manufacturing Supply Chain Regulatory Compliance, Specializing in Gene and Advances in Purification 9:45 Coffee Break in the Exhibit Hall with Tim Redmond, Director, Regional Sales, Sales, Cell Therapies Technologies Poster Viewing World Courier, Inc. The cell therapy supply chain needs expertise, 2:00 Identifying Stability-Indicating Virus & Pathogen Clearance TARGET PRODUCT PROFILES & which takes into account many elements. In this Parameters for Stability Studies and & Safety in Biologics presentation Tim Redmond will share some of the Beyond DEFINING QUALITY expertise that World Courier has garnered through Christopher Bravery, Ph.D., Consultant Regulatory ANALYTICAL & QUALITY the years in cell therapy logistics. 10:30 Use of Target Product Profile During Scientist, Consulting on Advanced Biologicals Ltd. Host Cell Proteins: Detection, • Learn how to plan ahead for the transport of Development This talk will address the need to identify stability- Analysis & Removal these critical shipments indicating parameters for the product and discuss Stefano Baila, Ph.D., Manager, Process • Understand regulatory requirements for cross how accelerated and/or forced degradation studies Early Analytical Development for Development, Celyad border shipments can be useful to achieve this. Considerations for the Biotherapeutics The Target Product Profile serves as the foundation • Gain knowledge on how to select the proper design and execution of a comprehensive stability Process Characterization, of the product and clinical development plan, with program and the use of stability data and stability- the final product properties and use in mind. This packaging for all phases of the supply chain – Qualification & Control from patient collection through to patient dosing indicating parameters beyond stability studies will approach takes all aspects into account, including also be discussed. FORMULATION & STABILITY quality, cost of goods, scalability and clinical use and • Understanding the Chain of Custody, and the Rapid Methods to Assess ensures alignment of all stakeholders during the importance of knowing where your shipments are 2:30 Release Testing Considerations for at all times Quality & Stability of Biologics development phases Different Cell Therapy Paradigms • Discover the role of specialized logistics to ensure Overcoming Formulation 11:00 A Multiparametric Approach for Joseph Sondej, Senior Manager, QC Operations, the personalized care of each patient’s samples Celgene Cellular Therapeutics Challenges Defining Product Quality and therapeutic agents Using examples this presentation will layout High-Concentration Damian Marshall, Ph.D., Head, Analytical 12:30 Go Beyond: How to Sponsored By considerations for testing fresh and frozen cell Protein Formulations Department, Cell and Gene Therapy Catapult therapy products as part of release testing Make Novel Therapies a The manufacture of cell therapy products can take considerations for different cell therapy paradigms. CELL & GENE THERAPY PRODUCTION several weeks or even months and involve a range Reality in Tomorrow’s Cell Therapy CMC, of complex processing steps. As a result it can Complex Biopharma Landscape 3:00 Mitigating Risk of Process Change: Quality & Analytics be challenging to define what cell characteristics Julie Murrell, Ph.D., Head, Cell Therapy Strategies and Tactics for Effective are most appropriate to measure in order to Bioprocessing, MilliporeSigma Comparability Cell Therapy Bioproduction, ensure product quality is being maintained. This A recent survey conducted by the Economist Scott Burger, M.D., Consultant, Advanced Cell and Operations & Logistics presentation will demonstrate a novel approach for Intelligence Unit highlights the new products that Gene Therapy marker screening and analysis that can be used the biopharma industry identifies as most disruptive Gene Therapy Bioproduction Manufacturing process changes are inevitable - to develop a framework to track product quality to their growth strategies in the next five years. processes are scaled up, methods and technology and to define the best approach for in-process The findings raise a challenge to biopharma to go Manufacturing & CMC are improved, raw materials are changed, operations control monitoring. beyond barriers to bringing new products to market Strategies for Biologics are transferred to new manufacturing sites. Any by presenting ways to overcome these hurdles and change to the manufacturing process, however, risks SPONSORS & EXHIBITORS make novel therapies a reality. altering the cell therapy product itself. This session HOTEL & TRAVEL will discuss methods, tactics, and requirements for prospectively managing process changes and REGISTRATION INFORMATION demonstrating comparability. BioprocessingSummit.com ◄ 49 ► COVER Cell & Gene Therapy Production EVENT AT A GLANCE Inaugural PLENARY KEYNOTE SESSION SHORT COURSES Cell Therapy CMC, Quality and Analytics TRAINING SEMINARS Understanding Your Product and Process for Late-Stage Success UPSTREAM PROCESSING Optimizing Cell Culture Technology 3:30 Refreshment Break in the Exhibit Hall Bioproduction: Scale, Bioreactors with Poster Viewing & Disposables COMPARABILITY & CMC STRATEGIES Optimizing Cell Line Development FOR CAR-Ts DOWNSTREAM PROCESSING 4:15 CAR-T Manufacturing and CMC Continuous Processing in Strategies Biopharm Manufacturing Nina Kotsopoulou, D.Phil., Director, Process Advances in Purification Development, Autolus Technologies Autolus is a biopharmaceutical company focused Virus & Pathogen Clearance on delivering CAR-T cell products to patients. This presentation will focus on the challenges of & Safety in Biologics combining efficient and fast delivery of Phase I/ ANALYTICAL & QUALITY II suitable processes and products, with ensuring timely delivery at Phase III (and with minimal Host Cell Proteins: Detection, comparability risk) of improved, robust processes Analysis & Removal suitable for commercialisation. Early Analytical Development for 4:45 Autologous Gene Therapy Biotherapeutics Manufacturing Comparability: Meeting the Process Characterization, Challenge through Process Understanding Qualification & Control and Product Characterization FORMULATION & STABILITY Michael Paglia, Ph.D., Senior Director, Technical Operations, Cellular Process Development and Rapid Methods to Assess Manufacturing, bluebird bio Quality & Stability of Biologics Demonstrating comparability of ex vivo autologous Overcoming Formulation gene therapy products and processes across Challenges multiple facilities in the US and EU may be required to meet capacity requirements. A comparability plan High-Concentration should therefore incorporate elements to support Protein Formulations both BLA and MAA as early in the development lifecycle as possible. A summary of comparability CELL & GENE THERAPY PRODUCTION approaches for ex vivo autologous gene therapy Cell Therapy CMC, products will be presented. Quality & Analytics 5:15 End of Conference Cell Therapy Bioproduction, Operations & Logistics 5:15 Registration for Dinner Short Course Gene Therapy Bioproduction Manufacturing & CMC Strategies for Biologics SPONSORS & EXHIBITORS HOTEL & TRAVEL REGISTRATION INFORMATION
BioprocessingSummit.com ◄ 50 ► COVER Cell & Gene Therapy Production EVENT AT A GLANCE 3rd Annual PLENARY KEYNOTE SESSION SHORT COURSES Cell Therapy Bioproduction, Operations and Logistics TRAINING SEMINARS Industrializing Cell Therapy Production for Commercial Manufacture UPSTREAM PROCESSING Optimizing Cell Culture Technology Bioproduction: Scale, Bioreactors WEDNESDAY, AUGUST 17 9:30 Preparing for Commercialization - The WORKING WITHIN CLOSED SYSTEMS & Disposables Orgenesis Story Optimizing Cell Line Development 7:00 am Registration Opens and Morning Vered Caplan, CEO, Orgenesis 1:45 Chairperson’s Remarks Coffee Ohad Karnieli, Ph.D., Chair, Process and Product DOWNSTREAM PROCESSING 10:00 Coffee Break in the Exhibit Hall with Development Committee, (ISCT) and CEO of Karnieli Continuous Processing in PREPARING FOR LATE-STAGE CELL Poster Viewing Ltd. Biopharm Manufacturing THERAPY MANUFACTURING SCALE-UP & SCALE-OUT STRATEGIES 1:50 New Technologies Driving A Closed Advances in Purification System Manufacturing Process 8:05 Chairperson’s Opening Remarks TO REDUCE COST OF GOODS Technologies Ohad Karnieli, Ph.D., Chair, Process and Product Ohad Karnieli, Ph.D., Chair, Process and Product Development committee, (ISCT) and CEO of Karnieli Virus & Pathogen Clearance Development committee, (ISCT) and CEO of Karnieli 10:45 Successful Cell Therapy Ltd. & Safety in Biologics Ltd Manufacturing: Think Big! As cell therapy matures moving form bed side Knut Niss, Ph.D., Asset Leader Cell Therapies, to clinic, the need for automated closed system ANALYTICAL & QUALITY 8:15 KEYNOTE PRESENTATION: Director, PO&T, Biogen manufacturing technologies become critical to Host Cell Proteins: Detection, Close logistical coordination is required from Applying Concepts of mAb and Vaccine insure quality, availability and cost efficiency of the obtaining the starting material to delivering the Analysis & Removal Manufacturing to Cellular Immune treatments. This need enhances dramatically in product (“bedside to bedside” or “arm-to-arm”). Early Analytical Development for Therapy cases of autologous therapies. The talk will describe While these challenges can be managed in early Alain Pralong, Ph.D., Senior Vice President, case studies of process development work and Biotherapeutics clinical trials through close interaction between the Manufacturing Operations, CellMedica present available and new technologies for closed manufacturing organization and the clinical site, Process Characterization, Massive evolution in cellular immune therapies and automated cell culturing systems. commercial needs require a focus on cost effective Qualification & Control for treatment of cancers has taken place over operations. Thus, to reach a commercially successful the last decade. A significant number of new 2:20 The Pros and Cons of Using Automated FORMULATION & STABILITY cell therapy, logistical challenges in combination companies has been created. Some have gained Closed Systems for Cell Therapy Trials with processing technologies need to be evaluated. Rapid Methods to Assess massive value through promising therapeutic Barbara Seymour, MBA, Senior Director, Quality & Stability of Biologics approaches. Manufacturing and GMP compliance 11:15 Scale-Up Strategies and Process Manufacturing, Unum Therapeutics Overcoming Formulation has not reached the maturity level of mAb and Optimisation for Cell Therapies Manufacturing autologous cell therapies in Vaccine manufacturing. How can proven concept automated closed systems may become industry Challenges Ravinder Bhatia, Ph.D., Director, Pharmaceutical be implemented in cellular immune therapy? standard, but many challenges can manifest when Development and Manufacturing Sciences, Johnson High-Concentration adapting a process to a system or vice versa. & Johnson Protein Formulations Labor savings and microbial reduction may be 9:00 Successful Cell Therapy Manufacturing: In this presentation, a case study will be presented offset by extended time in development to confirm CELL & GENE THERAPY PRODUCTION Think Big! on the considerations to select technologies to process robustness or the effects of any change. Knut_Niss Knut Niss, Ph.D., Asset Leader Cell develop a robust, and scalable process for allogeneic Cell Therapy CMC, Understanding system limitations, staffing, costs Therapies, Director, PO&T, Biogen cell therapy products. Also, a control strategy based Quality & Analytics and potential business continuity is essential for on QbD principles to consistently meet product Close logistical coordination is required from good clinical programs. Cell Therapy Bioproduction, obtaining the starting material to delivering the quality by controlling the raw materials attributes Operations & Logistics product (“bedside to bedside” or “arm-to-arm”). and process parameters will be discussed. 2:50 Transportating and Tracking Cell Gene Therapy Bioproduction While these challenges can be managed in early Therapies across Sites clinical trials through close interaction between the 11:45 Sponsored Presentations Professor Adrian Gee, Ph.D., Center for Cell & Gene manufacturing organization and the clinical site, (Opportunities Available) Manufacturing & CMC Therapy, Baylor College of Medicine Strategies for Biologics commercial needs require a focus on cost effective operations. Thus, to reach a commercially successful 12:15 Luncheon Presentation (Sponsorship As cellular therapies move towards licensure SPONSORS & EXHIBITORS cell therapy, logistical challenges in combination Opportunity Available) or Lunch on Your Own centralized manufacturing is inevitable. This with processing technologies need to be evaluated. necessitates development of optimal methods for HOTEL & TRAVEL 1:00 Session Break shipping starting cells and final products. Recent evidence suggests that functionality of some cells is REGISTRATION INFORMATION AUTOMATION, NEW TECHNOLOGIES & impaired by cryopreservation and that transportation of fresh cultures is preferable. In parallel, systems BioprocessingSummit.com ◄ 51 ► COVER Cell & Gene Therapy Production EVENT AT A GLANCE 3rd Annual PLENARY KEYNOTE SESSION SHORT COURSES Cell Therapy Bioproduction, Operations and Logistics TRAINING SEMINARS Industrializing Cell Therapy Production for Commercial Manufacture UPSTREAM PROCESSING Optimizing Cell Culture Technology Bioproduction: Scale, Bioreactors must be developed for tracking and tracing cells approaches to scale-up and commercialization. parts of an ever changing biotech landscape. But & Disposables from the time of collection to patient administration. sometimes these new companies are not equipped, 9:00 Developing a Scalable, Robust and Optimizing Cell Line Development or just cannot afford to pursue these ideas. They 3:20 Human Performance Improvement in Cost-Effective CAR-T Process have great ideas, but need some help. Sometimes DOWNSTREAM PROCESSING Cell Therapy Manufacturing and Quality Dawn Maier, Senior Manager, Cellular Process a more established institution with a trained staff Continuous Processing in Control Operations Development and Manufacturing, bluebird bio Inc. is needed. This is where off-site manufacturing comes in. Biopharm Manufacturing Gary du Moulin, Ph.D., MPH, RAC, Associate Adoptive transfer of autologous T cells genetically Professor, Massachusetts College of Pharmacy and engineered to express chimeric antigen receptors Advances in Purification Health Sciences University (CARs) has emerged as a promising approach for 11:45 Cellular immunotherapy: Product, Technologies the treatment of cancer. However, to fully realize the Process and Patents 3:50 Refreshment Break in the Exhibit Hall Virus & Pathogen Clearance therapeutic potential of these engineered T cells, David Brindley, Ph.D., Cooksey-Botnar-Saïd Fellow, with Poster Viewing simple and robust manufacturing processes that Saïd Business School/Paediatrics, University of & Safety in Biologics decrease time and cost are required. A summary Oxford 4:45 Plenary Keynote Session (see page 3 ANALYTICAL & QUALITY of the data to support bluebird bio’s approach and CAR-T immunotherapy efficacy is building, however for details) clinical platform will be presented. certain critical aspects of manufacturing processes Host Cell Proteins: Detection, remain largely unexplored, notably the impact of Analysis & Removal 6:00 Networking Reception in the Exhibit 9:30 Sponsored Presentations (Opportunities leachables and extractables. Combined with a Early Analytical Development for Hall with Poster Viewing Available) complex patent landscape and a shift away from patenting the therapeutic entity towards patents of Biotherapeutics 7:00 End of Day 10:00 Coffee Break in the Exhibit Hall with processes and components, a number of pertinent Process Characterization, Poster Viewing challenges for both academia and industry exist. Qualification & Control THURSDAY, AUGUST 18 OFF-THE-SHELF/ OFF-SITE CAR-T 12:15 Lunch Available for Purchase in the FORMULATION & STABILITY 8:00 am Registration Opens and Morning MANUFACTURING Exhibit Hall Rapid Methods to Assess Coffee Quality & Stability of Biologics 10:45 Manufacturing Cellectis’ Allogeneic 1:15 Dessert Refreshment Break in the Exhibit Hall with Poster Viewing Overcoming Formulation CAR-T MANUFACTURING STRATEGIES UCART Product Candidates Sylvain Arnould, Head, Manufacturing, Cellectis Challenges 8:25 Chairperson’s Remarks 1:55 End of Conference At Cellectis, we are manufacturing allogeneic High-Concentration Ohad Karnieli, Ph.D., Chair, Process and Product off-the-shelf UCART-cell product candidates. The Protein Formulations Development Committee, (ISCT) and CEO of Karnieli specificity of those allogeneic therapies is that Ltd. CELL & GENE THERAPY PRODUCTION T-cells from healthy donors are genetically edited 8:30 Commercialization of ex vivo Cell with our proprietary technology TALEN®. This Cell Therapy CMC, approach could lead to a cost-effective drug, easily Quality & Analytics and Gene Therapy Products: Establishing distributed across all geographies and available to Processes, Manufacturing and Supply Cell Therapy Bioproduction, cancer patients. Chains Operations & Logistics Bo Kara, Head, Process Development, Cell Gene 11:15 Off-Site Manufacturing Challenges Gene Therapy Bioproduction Therapy, Platform CMC, GSK when Dealing with CAR-T Cells The complexities of the CGT supply chain Sean Smith, Ph.D., Cell Processing Specialist, Dana- Manufacturing & CMC requires cost sensitivity analysis driven process Farber Cancer Institute Strategies for Biologics development for cost efficient delivery of plasmid Off site manufacturing is a viable option for some SPONSORS & EXHIBITORS DNA’s, vector manufacturing and cell processing. companies producing cell therapies, but like any This presentation will discuss the technology and other process, it has its pros and cons. New start HOTEL & TRAVEL process development challenges in the delivery of up companies. New ideas. Both are a important REGISTRATION INFORMATION ex-vivo cell gene therapy medicines and describe
BioprocessingSummit.com ◄ 52 ► COVER Cell & Gene Therapy Production EVENT AT A GLANCE Inaugural PLENARY KEYNOTE SESSION SHORT COURSES Gene Therapy Bioproduction and CMC TRAINING SEMINARS Process Development, Scale-Up and Analysis of Gene Therapies UPSTREAM PROCESSING Optimizing Cell Culture Technology THURSDAY, AUGUST 18 3:15 Lentiviral Vector Optimization 5:15 End of Day Strategies for Higher LV Production and Bioproduction: Scale, Bioreactors 11:30 am Registration Opens Safety 5:15 Registration for Dinner Short Course & Disposables 12:15 pm Lunch Available for Purchase in Dmitriy Lukashev, Ph.D., Principal Scientist, Novartis Optimizing Cell Line Development Institutes for BioMedical Research FRIDAY, AUGUST 19 the Exhibit Hall Using case studies, this presentation discusses DOWNSTREAM PROCESSING 8:00 am Registration Opens and Morning 1:15 Dessert Refreshment Break in the rationale for changes in lentiviral vector to increase Continuous Processing in Coffee Exhibit Hall with Poster Viewing infectious titer and improve safety; Comparison of Biopharm Manufacturing various titer methods for evaluation of LV titers in Advances in Purification process development; Significance of transfer vector CMC, QUALITY & POTENCY ASSAY PROCESS DEVELOPMENT STRATEGIES cis-elements in production of lentiviral vectors. Technologies FOR LENTIVIRUS-BASED PROCESSES DEVELOPMENT Virus & Pathogen Clearance 3:45 Sponsored Presentation (Opportunity 8:25 Chairperson’s Remarks 1:55 Chairperson’s Opening Remarks & Safety in Biologics Available) Michael Kelly, Ph.D., Director, Asset Leadership, 8:30 Gene Therapies for All: How to ANALYTICAL & QUALITY Gene Therapy, Biogen 4:00 Refreshment Break Approach Industrialization of Gene Host Cell Proteins: Detection, Therapies on a Large Scale 2:00 KEYNOTE PRESENTATION: Analysis & Removal MANUFACTURING, COMPARABILITY & Ben Locwin, Ph.D., MBA, MS, President, Healthcare Optimising the Production of TECH TRANSFER Science Advisors Early Analytical Development for Lentiviruses Biotherapeutics While precision medicine, personalized medicine, Steven Howe, Ph.D., Head, Process Research, 4:15 Lentiviral Vector Manufacturing and gene therapies represent the future of targeted Process Characterization, Cell & Gene Therapy Platform CMC, GSK Strategies to Support Cell and Gene therapies for improved disease management and Qualification & Control This presentation will discuss the progress Therapies resolution, there are challenges of scale that must made in retroviral vectorology in recent years be faced in order to make GT viable for society. In FORMULATION & STABILITY Carol Knevelman, Ph.D., Senior Manager, Process and now that gene therapy is a clinical reality, R&D Department, Oxford Biomedica Ltd. this engaging discussion about the future of GT, best Rapid Methods to Assess new challenges and opportunities associated Successful development and commercialization practices will be shown to increase the likelihood Quality & Stability of Biologics with producing lentiviral viruses on a commercial of gene and cell-based therapies is highly that gene therapies can successfully represent the scale. Understanding and refining these next generation of medicine. Concepts covered dependent on establishing robust and efficient Overcoming Formulation processes will lead to more cost-effective will include Quality Risk Management, Quality by Challenges cGMP manufacturing processes that meet the key manufacture of vectors to enable wider challenges of scale, quality, cost of goods supplied Design, CPPs, and CQAs. High-Concentration application of gene therapy. (COGS), and sustainability. This talk will discuss new 9:00 Analytics for AAV Gene Therapy, Protein Formulations manufacturing modalities that are being developed 2:45 Enabling Industrial Scale Production of for cGMP production of these vectors. Strategies for Phase I CELL & GENE THERAPY PRODUCTION Lentiviral Vectors for Gene Therapy Genine Winslow, Ph.D., Director of Research Cell Therapy CMC, Kelly Kral, Ph.D., Senior Manager, Vector Process 4:45 Demonstrating Comparability during Analytics, Audentes Therapeutics, Inc. Quality & Analytics Development and Manufacturing, bluebird bio Gene Therapy Tech Transfer Using case studies this presenation will look at an Lentiviral vectors are an ideal platform for indications overview of CMC requirements for phase 1 CTA/IND Cell Therapy Bioproduction, Christopher Bravery, Ph.D., Consultant Regulatory requiring long-term, stable expression, but the Scientist, Consulting on Advanced Biologicals Ltd. applications, AAV CMC analytics challenges with Operations & Logistics production processes have historically been Changing the manufacturing site (tech transfer) emphasis on achieving comparable vector genome Gene Therapy Bioproduction limited by scale. This presentation will focus on should always include an assessment of titers across sites, and a brief discussion of the evaluation of platforms for the next-generation comparability, however the ability to demonstrate potency assays. The talk will also look at strategies Manufacturing & CMC manufacturing process, with the guiding principles this varies between early and late development. This for successful coordination of AAV analytics Strategies for Biologics of preserving the comparability of the lentiviral talk will discuss common pitfalls and mistakes and with CMOs. product profile. SPONSORS & EXHIBITORS highlight key aspects of the comparability exercise. HOTEL & TRAVEL REGISTRATION INFORMATION
BioprocessingSummit.com ◄ 53 ► COVER Cell & Gene Therapy Production EVENT AT A GLANCE Inaugural PLENARY KEYNOTE SESSION SHORT COURSES Gene Therapy Bioproduction and CMC TRAINING SEMINARS Process Development, Scale-Up and Analysis of Gene Therapies UPSTREAM PROCESSING Optimizing Cell Culture Technology 9:30 Developing a Potency Assay Matrix for 11:30 From Initial Phase I Sponsored By 2:30 Gene and Cell Therapy for Inherited Bioproduction: Scale, Bioreactors an AAV Gene Therapy Product Supply with CRO to Phase Diseases: Cystic Fibrosis and Sickle Cell & Disposables Barb Thorne, Ph.D., Consultant, Thorne Bio- III CMO Supply Anemia Consulting LLC, Former Executive Director Process Optimizing Cell Line Development Jean-Philippe Combal, Ph.D., COO, GenSight We have developed protocols for efficiently Development, Celladon Biologics, France correcting disease associated mutations in the DOWNSTREAM PROCESSING A case study will be presented on the potency Jolyn Johnson, Ph.D., Upstream Processing Expert, CF transmembrane conductance regulator (CFTR) Continuous Processing in matrix for a gene therapy product formerly under Novasep and human beta-globin using CRISPR/Cas9 and development, AAV1/SERCA2a. The approach TALEN to enhance polynucleotide gene editing. Biopharm Manufacturing More rigorous product characterization, process was based on FDA Guidance on Potency Tests development and regulatory hurdles for phase 3 or We have developed protocols for assessment Advances in Purification for Cellular and Gene Therapy Products, and the even commercial batches require highly skilled and of efficient clonal isolation of corrected iPS cells Technologies matrix included three complementary quantitative critical transfer. Challenges and anticipation from and their directed differentiation into airway assays used for product lot release and stability and hematopoietic progenitor cells to promote Virus & Pathogen Clearance rights to know-how transfer and regulatory impact testing as well as a challenging qualitative enzyme requires significant perspectives to ensure large- efficacious therapeutic transplantation. & Safety in Biologics activity assay. scale cGMP and regulatory compliance. Viewpoint from both biotech and cGMP will be discussed. 3:00 Panel Discussion: Commercializing ANALYTICAL & QUALITY 10:00 Networking Coffee Break Gene Therapies Host Cell Proteins: Detection, 12:00 pm Sponsored Presentations Panelists: Analysis & Removal PROCESS DEVELOPMENT FOR AAV- (Opportunities Available) David Dismuke, Director, Vector Production, Voyager Early Analytical Development for BASED PROCESSES Therapetuics 12:30 Luncheon Presentation (Sponsorship Biotherapeutics Jeffrey Bartlett, Ph.D., Senior Vice President, R&D, 10:30 A Commercially Viable Novel CMC Opportunity Available) or Lunch on Your Own Calimmune Process Characterization, Approach to Bioprocessing AAV for Rare 1:15 Session Break Dieter Gruenert, Ph.D., Professor, Department of Qualification & Control Diseases Otolaryngology, Head and Neck Surgery, University FORMULATION & STABILITY Dr Reed Clark, Sr VP Mfg, Dimension Therapeutics PROCESS DEVELOPMENT of California This presentation will look at creating a continuum Topics to be covered include: Rapid Methods to Assess for both upstream and downstream, viewing large & SCALE-UP STRATEGIES • Pricing - from process to reimbursement Quality & Stability of Biologics scale manufacture from the very beginning and the 1:25 Chairperson’s Remarks • Scaling up - unit operations, platforms, analytics Overcoming Formulation main challneges associated with cost and scale-up. • Gene therapy supply - from rare disease to Challenges 1:30 rAAV Vector Production in the 11:00 Late Breaking Presentation larger indications High-Concentration Baculovirus/Sf9 platform and in HEK293 • Facility design Protein Formulations Suspension Cell System David Dismuke, Director, Vector Production, Voyager 3:30 Close of Conference CELL & GENE THERAPY PRODUCTION Therapetuics Cell Therapy CMC, Quality & Analytics 2:00 Strategies to Deliver Scalable and Reliable Lentiviral Vector Biomanufacturing Cell Therapy Bioproduction, Jeffrey Bartlett, Ph.D., Senior Vice President, R&D, Operations & Logistics Calimmune Gene Therapy Bioproduction One important consideration in designing processes for the clinical production of lentiviral vectors is Manufacturing & CMC minimizing lot-to-lot variation. With a mind towards Strategies for Biologics developing a scalable, reliable, cGMP-compliant biomanufacturing process for lentiviral vectors SPONSORS & EXHIBITORS we have established the Cytegrity stable cell line HOTEL & TRAVEL system and have defined key regulatory parameters to facilitate cGMP production and regulatory REGISTRATION INFORMATION approval using this system. BioprocessingSummit.com ◄ 54 ► COVER EVENT AT A GLANCE Inaugural PLENARY KEYNOTE SESSION SHORT COURSES Manufacturing and CMC Strategies for Biologics TRAINING SEMINARS Flexible Manufacturing and CMC Strategies for Emerging and Legacy Products UPSTREAM PROCESSING Optimizing Cell Culture Technology MONDAY, AUGUST 15 2:15 Setting Standards in Single-Use 4:30 Breakout Discussions Bioproduction: Scale, Bioreactors Systems: Update from SUTAP, The Single- 8:00 am Short Course Registration Use Technology Assessment Program 5:30 Grand Opening Reception in the & Disposables Exhibit Hall with Poster Viewing Alain Pralong, Ph.D., Senior Vice President, Optimizing Cell Line Development 9:00 – 11:30 Recommended Morning Manufacturing Operations, CellMedica 7:00 End of Day DOWNSTREAM PROCESSING Short Course* The shift from traditional to single-use technology New USP Initiatives and Standards for Biologics Continuous Processing in is changing the distribution of responsibility and * Separate registration required, see page 5 for details. accountability within the drug manufacturing value TUESDAY, AUGUST 16 Biopharm Manufacturing chain as more components are procured ready-to- Advances in Purification 11:30 Main Conference Registration Opens use without further modification by the end-user 7:30 am Registration Opens and Morning Technologies meaning the effective drug manufacturer. This Coffee SINGLE-USE MANUFACTURING & presentation will outline how the creation of SUTAP Virus & Pathogen Clearance FACILITY DESIGN – the single-use technology assessment program – PRODUCT LIFECYCLE MANAGEMENT & Safety in Biologics has progressed, where we stand today and give a & CONTINUOUS PROCESS comprehensive overview on the next steps. ANALYTICAL & QUALITY 1:00 pm Chairperson’s Opening Remarks IMPROVEMENT Jeff Odum, Global Technology Partner, NNE Host Cell Proteins: Detection, 2:45 Refreshment Break Pharmaplan 7:55 Chairperson’s Remarks Analysis & Removal 1:10 Single Use Technologies and MANUFACTURING STRATEGIES FOR Franqui Jimenez, Ph.D., Senior Director, Head of Early Analytical Development for Manufacturing Science and Technology, Genzyme Biotherapeutics Implementation for Biomanufacturing: The NOVEL BIOLOGICS Design Manager’s Challenge 8:00 Application of Advanced Process Characterization, Jeff Odum, Global Technology Partner, NNE 3:15 Analytical Testing Strategy to Support Manufacturing Process Strategies Qualification & Control Pharmaplan Kadcyla® / T-DM1 Manufacturing Process and Analytical Tools for Increased Validation for Supply Chain Optimization FORMULATION & STABILITY As companies develop both short and long-term Process Throughput, Improved Product manufacturing strategies, operational efficiency Lan Dai, Ph.D., Associate QA Scientist, Global Understanding, and Efficient Process Rapid Methods to Assess Biologics Quality Control, Genentech/Roche and utilization become very critical to optimizing Control Quality & Stability of Biologics cost-of-goods, manpower planning, and supply Cleaning Validation is a key step to de-bottleneck Valerie Tsang, Ph.D., Senior Engineer III, Technical chain management. This article will provide insights during manufacturing site transfer. The complexity Overcoming Formulation Development, Biogen Challenges into the challenges faced by Design Managers is increased for ADC product where the cyctotoxic implementing new single-use technology platforms residuals must be removed to be below a pre- In order to meet the potentially high commercial High-Concentration and the sometimes unforeseen issues they face. determined Maximum Allowable Carryover level demand for some biologics programs, Protein Formulations The topics will include defining the process and after equipment cleaning. A holistic approach manufacturing process changes are often optimizing time-and motion analysis, equipment has been used in designing the QC testing/ necessary during the late stages of clinical product CELL & GENE THERAPY PRODUCTION definition and platform technology analysis. validation strategy, including considerations to link development. To accommodate the anticipated Cell Therapy CMC, downstream analytical testing to the particular market needs while maintaining consistently high Quality & Analytics 1:45 KEYNOTE PRESENTATION: cleaning process and to ensure compliance in quality products comparable to those used in early phase clinical studies, Biogen has developed and Cell Therapy Bioproduction, Developing Practical Single-Use accordance to local CMO and company network practice. implemented a series of process technologies Operations & Logistics Processes for New Vaccine Formulations and analytical tools to increase productivity and Kirsten Strahlendorf, P.Eng, M.Eng, Senior Gene Therapy Bioproduction 4:00 An Integrated Platform for Mass throughput, improve process consistency and Scientist, Sanofi Pasteur Ltd. Production of Immunotherapy Applications product understanding. Manufacturing & CMC In order to successfully implement a new vaccine Robert Dream, Ph.D., Consultant 8:30 Emerging Process Trends in Biologics Strategies for Biologics formulation process using closed system, single-use technologies, both end-user process Thus far using gene-modified cells has mainly been Development and Manufacturing carried out by investigators who have developed SPONSORS & EXHIBITORS and study design require careful thought and Kumar Dhanasekharan, Ph.D., Director of Process their manufacturing process for small scale clinical execution. This presentation will review some Development, Cook Pharmica LLC HOTEL & TRAVEL of the aspects of filtration, mixing, formulating trials by using the devices and infrastructure at hand. Anyone who has embarked on the task of REGISTRATION INFORMATION and dispensing that can impact homogeneity, adsorption, concentration and other critical manufacturing patient-specific advanced therapeutic medicinal products (ATMP) for clinical use will BioprocessingSummit.com product attributes. ◄ 55 ►
admittedly agree that it is quite an undertaking. COVER EVENT AT A GLANCE Inaugural PLENARY KEYNOTE SESSION SHORT COURSES Manufacturing and CMC Strategies for Biologics TRAINING SEMINARS Flexible Manufacturing and CMC Strategies for Emerging and Legacy Products UPSTREAM PROCESSING Optimizing Cell Culture Technology 9:00 Considerations for Implementing a 11:30 CMC Challenges in Practice: 2:30 Accelerating Timeline to IND by Using Bioproduction: Scale, Bioreactors Development Laboratory to Support a Comparability vs. Similarity Pool for Tox Strategy & Disposables Legacy Product Beatrix Metzner, Ph.D., Director, Global CMC Wendy Hsu, Engineer and Group Leader, Early Optimizing Cell Line Development Gregory Walsh, M.S. Associate Director Strategy & Tech RA, Boehringer Ingelheim Pharma Stage Cell Culture, Genentech Manufacturing Sciences and Technology Laboratory, GmbH & Co. KG Speed to IND submission is essential in establishing DOWNSTREAM PROCESSING Genzyme The impact of manufacturing changes is a competitive advantage for a therapeutic product. Continuous Processing in When improving upon a legacy process an well understood by means of comparability CMC activities are often on the critical path to IND- Biopharm Manufacturing understanding of the history and the past exercises. Mostly analytical testing is sufficient. enabling toxicology (Tox) studies. The use of a “Pool The establishment of biosimilarity requires for Tox” strategy where a pool of clonally derived Advances in Purification regulatory strategy of the product should be well understood. However not all legacy products have a comprehensive analytical comparability cell lines is used instead of a single, clonally derived Technologies all the information available that meet current best exercise very sensitive to differences and allows cell line to generate Tox material, can accelerate Virus & Pathogen Clearance practices. Lifecycle management of legacy products determining if a proposed biosimilar is shown timelines by several months. The success of the & Safety in Biologics require continual process knowledge building, to be “highly similar”. Therefore the scientific strategy relies on the ability to generate comparable manufacturing support, and process improvements principle of a biosimilarity exercise is based on the Phase I material using a single, clonally derived ANALYTICAL & QUALITY which may require regulatory filings. This case study comparability approach. cell line. presents the implementation of a development Host Cell Proteins: Detection, 12:00 pm Sponsored Presentations 3:00 A Client’s Perspective- Developing an Analysis & Removal laboratory solely dedicated to a legacy commercial product that is capable of support all aspects of (Opportunities Available) Accelerated Timeline to File an IND Early Analytical Development for manufacturing and analytics. Deborah Meshulam, Ph.D., Director, Contract Biotherapeutics 12:30 Luncheon Presentation (Sponsorship Manufacturing, Scholar Rock 9:30 Sponsored Presentation (Opportunity Opportunity Available) or Lunch on Your Own Process Characterization, Scholar Rock has developed proprietary technology Available) Qualification & Control 1:15 Dessert Refreshment Break in the based on new insights into the structural biology and activation mechanisms of protein 9:45 Coffee Break in the Exhibit Hall with Exhibit Hall with Poster Viewing FORMULATION & STABILITY growth factors. The process of developing and Poster Viewing Rapid Methods to Assess manufacturing antibodies which can modulate CMC STRATEGIES TO ACCELERATE these growth factors can be accelerated to file Quality & Stability of Biologics BIOSIMILARS: MANUFACTURING & TOWARDS IND an IND using a GPEx technology cell line. In this Overcoming Formulation CMC STRATEGIES presentation, key parameters and potential risks will Challenges 1:55 Chairperson’s Remarks be identified and discussed. 10:30 Biosimilars Manufacturing Strategies: High-Concentration 2:00 Regulatory CMC for Biosimilars: The Can New Entities, CMOs and Others 3:30 Refreshment Break in the Exhibit Hall Protein Formulations Comparability Exercise Effectively Compete with Big Bio/Pharma? with Poster Viewing Mr Richard DiCicco, Harvest Moon Pharmaceuticals CELL & GENE THERAPY PRODUCTION Eric S. Langer, President and Managing Partner, USA Inc IMPROVING MANUFACTURING, Cell Therapy CMC, BioPlan Associates For the approval of biosimilars, a meaningful FORMULATION & PRODUCT DESIGN Quality & Analytics 11:00 Choosing between Patent and Trade “Finger-Print”-like analysis algorithm is needed to Cell Therapy Bioproduction, Secret to Protect Bioproduction Methods demonstrate similarity of the biosimilar product to 4:15 Novel Stability and Concentration the Reference Product. This presentations looks Operations & Logistics Paul Calvo, Ph.D., Director, Biotechnoogy Group, Enhancing Formulations for Improved at a stepwise approach starting with analytics to Sterne, Kessler, Goldstein & Fox P.L.L.C. Administration of Biotherapeutics Gene Therapy Bioproduction prove highly similar physio-chemical attributes, With estimates that approximately $65 billion worth batch-to-batch variability of the quality attributes Neil Schauer, Ph.D., Advisor, ReForm Biologics Manufacturing & CMC of biologics will be coming off patent by 2020, the of the Reference Product and analytical structural The current trend in biologic drug delivery is towards Strategies for Biologics choice of trade secrets versus patent protection has characterisation for biosimilar comparability. development of formulations that enable room taken on renewed importance in the biotechnology temperature stable, high protein concentration drug SPONSORS & EXHIBITORS sector. This talk will dicuss choosing between patent products in prefilled syringes and auto injectors. HOTEL & TRAVEL and trade secret to protect bioproduction methods. This trend requires novel formulation strategies. This presentation addresses product viscosity and REGISTRATION INFORMATION stability issues for the improved administration of biotherapeutics. BioprocessingSummit.com ◄ 56 ► COVER
EVENT AT A GLANCE Inaugural PLENARY KEYNOTE SESSION SHORT COURSES Manufacturing and CMC Strategies for Biologics TRAINING SEMINARS Flexible Manufacturing and CMC Strategies for Emerging and Legacy Products UPSTREAM PROCESSING Optimizing Cell Culture Technology 4:45 Facilitating the Design of Recombinant Bioproduction: Scale, Bioreactors Protein Therapeutics with the Aid of & Disposables Comprehensive Genomic Codon Usage Optimizing Cell Line Development Data Chava Kimchi-Sarfaty, Ph.D., Principal Investigator, DOWNSTREAM PROCESSING Hematology, FDA/CBER Continuous Processing in Codon usage is an active area of research that Biopharm Manufacturing plays a major role in the expression and folding Advances in Purification of proteins, and is especially important in the creation of recombinant therapeutics. However, Technologies genomic codon usage data is outdated, inaccurate, Virus & Pathogen Clearance or unavailable in existing databases for many & Safety in Biologics organisms, limiting opportunities for research. We are creating a new database to provide codon usage ANALYTICAL & QUALITY data for every available organism. Host Cell Proteins: Detection, 5:15 End of Conference Analysis & Removal Early Analytical Development for 5:15 Registration for Dinner Short Course Biotherapeutics 6:00 – 8:30 Recommended Dinner Short Process Characterization, Course* Qualification & Control Analytical Strategies for Comparability in FORMULATION & STABILITY Bioprocess Development * Separate registration required, see page 5 for details. Rapid Methods to Assess Quality & Stability of Biologics Overcoming Formulation Challenges High-Concentration Protein Formulations CELL & GENE THERAPY PRODUCTION Cell Therapy CMC, Quality & Analytics Cell Therapy Bioproduction, Operations & Logistics Gene Therapy Bioproduction Manufacturing & CMC Strategies for Biologics SPONSORS & EXHIBITORS HOTEL & TRAVEL REGISTRATION INFORMATION
BioprocessingSummit.com COVER EVENT AT A GLANCE PLENARY KEYNOTE SESSION SPONSORSHIP & EXHIBITOR OPPORTUNITIES SHORT COURSES CHI offers comprehensive packages that can be customized to your budget and objectives. Sponsorship allows TRAINING SEMINARS you to achieve your goals before, during, and long after the event. Packages may include presentations, exhibit UPSTREAM PROCESSING space and branding, as well as the use of delegate lists. Signing on early will maximize your exposure to qualified Optimizing Cell Culture Technology decision-makers and drive traffic to your website in the coming months. Bioproduction: Scale, Bioreactors & Disposables Podium Presentations — Available within Main Agenda! Exhibit Optimizing Cell Line Development Showcase your solutions to a guaranteed, targeted audience through Exhibitors will enjoy facilitated networking opportunities with qualified a 15- or 30-minute presentation during a specific conference program, delegates, making it the perfect platform to launch a new product, collect DOWNSTREAM PROCESSING breakfast, lunch, or separate from the main agenda within a pre- feedback, and generate new leads. Exhibit space sells out quickly, so Continuous Processing in conference workshop. Package includes exhibit space, on-site branding, reserve yours today! Biopharm Manufacturing and access to cooperative marketing efforts by CHI. For the luncheon Advances in Purification option, lunches are delivered to attendees who are already seated in the Additional branding and promotional opportunities are Technologies main session room. Presentations will sell out quickly, so sign on early to available, including: Virus & Pathogen Clearance secure your talk! • Conference Tote Bags & Safety in Biologics • Literature Distribution (Tote Bag Insert or Chair Drop) Invitation-Only VIP Dinner/Hospitality Suite ANALYTICAL & QUALITY Select specific delegates from the pre-registration list to attend a private • Badge Lanyards Host Cell Proteins: Detection, function at an upscale restaurant or a reception at the hotel. From • Program Guide Advertisement Analysis & Removal extending the invitations, to venue suggestions, CHI will deliver your • Padfolios and More... Early Analytical Development for prospects and help you make the most of this invaluable opportunity. Biotherapeutics Process Characterization, Focus Group For more information, please contact: Qualification & Control CHI will gladly provide you the opportunity of running a focus group Companies A-K Companies L-Z FORMULATION & STABILITY on-site. This exclusive gathering can be useful to conduct market Sherry Johnson Carolyn Benton Rapid Methods to Assess research, collect feedback on a new product idea, and collect marketing Business Development Manager Business Development Manager Quality & Stability of Biologics intelligence from industry experts on a specific topic. 781-972-1359 781-972-5412 [email protected] [email protected] Overcoming Formulation Challenges User Group Meeting/Custom Event Co-locate your user group meeting or custom event. CHI will help market High-Concentration the event, manage logistical operations, develop the agenda, and more. Protein Formulations CHI can handle the entirety of the meeting or select aspects. CELL & GENE THERAPY PRODUCTION Cell Therapy CMC, Quality & Analytics 2016 Sponsors & Exhibitors Applied Photophysics Eppendorf North America MilliporeSigma SensiQ Technologies Inc. Cell Therapy Bioproduction, Aragen Essential Pharmaceuticals, LLC NanoTemper Technologies, Inc. Solentim Operations & Logistics Asahi Kasei Bioprocess America, Inc. Eurofins Lancaster Laboratories Nova Biomedical Soluble Therapeutics Gene Therapy Bioproduction Analytical Technologies Group Finesse NSF Health Sciences Texcell - North America, Inc. Avid Bioservices, Inc. Flownamics Analytical Instruments, Inc. Pall Life Sciences Thomson Instrument Company Manufacturing & CMC BioGenes Genedata Pfanstiehl Toxikon Corporation Strategies for Biologics Bio-Rad Laboratories GYROS, Inc. ProteinSimple Tosoh Bioscience LLC SPONSORS & EXHIBITORS Bruker Corporation Hamilton Company Prozyme, Inc. Unchained Labs Caprion Infors USA Inc. Purolite Wako USA HOTEL & TRAVEL ChemoMetec, Inc. JSR Life Sciences Rentschler Biotechnologie GmbH WuXi AppTec REGISTRATION INFORMATION Corning Incorporated Kuhner Shaker RheoSense, Inc. Cygnus Technologies Lonza S-BIO, Sumitomo Bakelite Co., Ltd. BioprocessingSummit.com Distek, Inc. Malvern Instruments Sartorius Stedim Biotech ◄ 58 ► COVER EVENT AT A GLANCE PLENARY KEYNOTE SESSION HOTEL & TRAVEL INFORMATION SHORT COURSES TRAINING SEMINARS Conference Venue and Hotel: Reservations: Media Partners UPSTREAM PROCESSING Please visit the Travel page of Westin Boston Waterfront Lead Sponsoring Publications: Optimizing Cell Culture Technology 425 Summer St. www.bioprocessingsummit.com Bioproduction: Scale, Bioreactors Boston, MA 02210 for further details and to book your hotel. & Disposables Phone: 617-532-4600 Optimizing Cell Line Development Discounted Room Rate: $249 s/d DOWNSTREAM PROCESSING Discounted Cut-off Date: July 20, 2016 Continuous Processing in Sponsoring Publications: Biopharm Manufacturing Advances in Purification Top Reasons to Stay at the Westin Boston Waterfront Hotel FierceBiotech Technologies THE BIOTECH INDUSTRY’S DAILY MONITOR • Complimentary Wireless Internet in • Lots of new restaurants within walking Virus & Pathogen Clearance your guest room distance, Waterfront is the up-and- & Safety in Biologics • Take advantage of the deeply coming area of Boston! ANALYTICAL & QUALITY discounted $249 group rate! • No Commute - conference takes place Host Cell Proteins: Detection, at the hotel Analysis & Removal Web Partners: Early Analytical Development for Biotherapeutics Process Characterization, Qualification & Control FORMULATION & STABILITY Rapid Methods to Assess Quality & Stability of Biologics Overcoming Formulation Challenges High-Concentration Protein Formulations CELL & GENE THERAPY PRODUCTION Cell Therapy CMC, Quality & Analytics Cell Therapy Bioproduction, Operations & Logistics Gene Therapy Bioproduction Manufacturing & CMC Strategies for Biologics SPONSORS & EXHIBITORS HOTEL & TRAVEL REGISTRATION INFORMATION
BioprocessingSummit.com ◄ 59 ► COVER EVENT AT A GLANCE Eighth Annual PLENARY KEYNOTE SESSION SHORT COURSES THE SUMMIT TRAINING SEMINARS BIOPROCESSING UPSTREAM PROCESSING Optimizing Cell Culture Technology Practical Solutions for Today’s Bioprocess Challenges Bioproduction: Scale, Bioreactors August 15-19, 2016 | Westin Boston Waterfront , Boston, MA & Disposables Optimizing Cell Line Development DOWNSTREAM PROCESSING Academic, Government, Continuous Processing in Commercial Hospital-affiliated Biopharm Manufacturing CONFERENCE & TRAINING SEMINAR PRICING CONFERENCE DISCOUNTS Advances in Purification Technologies PREMIUM PACKAGE (Includes access to all conferences and training seminars Monday-Friday. Excludes short courses.) Poster Submission - Discount ($50 Off): Poster abstracts are due by July 15, 2016. Once your Virus & Pathogen Clearance Special Early-Bird Registration Deadline until April 22, 2016 $2595 $1375 registration has been fully processed, we will send & Safety in Biologics Early Registration Deadline until June 10, 2016 $2845 $1455 an email containing a unique link allowing you to ANALYTICAL & QUALITY Advance Registration Deadline until July 15, 2016 $3045 $1555 submit your poster abstract. If you do not receive your link within 5 business days, please contact jring@ Host Cell Proteins: Detection, Registrations after July 15, 2016 and on-site $3245 $1625 healthtech.com. *CHI reserves the right to publish Analysis & Removal STANDARD PACKAGE (Includes access to two conferences and/or training seminars. Excludes short courses) your poster title and abstract in various marketing Early Analytical Development for materials and products. Special Early-Bird Registration Deadline until April 22, 2016 $2195 $1125 Biotherapeutics REGISTER 3 - 4th IS FREE: Individuals must register for Early Registration Deadline until June 10, 2016 $2295 $1225 Process Characterization, the same conference or conference combination and submit Qualification & Control Advance Registration Deadline until July 15, 2016 $2495 $1295 completed registration form together for discount to apply. Registrations after July 15, 2016 and on-site $2695 $1395 FORMULATION & STABILITY Group Discounts: Discounts are available for multiple BASIC PACKAGE (Includes access to one conference or training seminar. Excludes short courses) attendees from the same organization. For more information Rapid Methods to Assess on group rates contact Elizabeth Lemelin: Quality & Stability of Biologics Special Early-Bird Registration Deadline until April 22, 2016 $1445 $625 1-781-972-5488 | [email protected] Overcoming Formulation Early Registration Deadline until June 10, 2016 $1575 $705 If you are unable to attend but would like to purchase The Challenges Advance Registration Deadline until July 15, 2016 $1725 $785 Bioprocessing Summit CD for $750 (plus shipping), please High-Concentration Registrations after July 15, 2016 and on-site $1925 $855 visit BioprocessingSummit.com. Massachusetts delivery will Protein Formulations include sales tax. CELL & GENE THERAPY PRODUCTION SHORT COURSE PRICING ADDITIONAL REGISTRATION DETAILS Cell Therapy CMC, One short course $595 $345 Quality & Analytics Each registration includes all conference sessions, posters and Two short courses $895 $595 exhibits, food functions, and access to the conference proceedings Cell Therapy Bioproduction, Three short courses $1095 $695 link. Operations & Logistics Handicapped Equal Access: In accordance with the ADA, Cambridge Gene Therapy Bioproduction Healthtech Institute is pleased to arrange special accommodations for attendees with special needs. All requests for such assistance must be submitted in writing to CHI at least 30 days prior to the start Manufacturing & CMC of the meeting. Strategies for Biologics How to Register: BioprocessingSummit.com To view our Substitutions/Cancellations Policy, go to SPONSORS & EXHIBITORS www.healthtech.com/regdetails [email protected] • P: 781.972.5400 or Toll-free in the U.S. 888.999.6288 HOTEL & TRAVEL Please use keycode 1681 F when registering Video and or audio recording of any kind is prohibited onsite at all CHI events. REGISTRATION INFORMATION
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