Post-Event Summary

Total Page:16

File Type:pdf, Size:1020Kb

Post-Event Summary VIRTUAL BIOMANUFACTURING WORLD SUMMIT 20 NOVEMBER 16-17, 2020 POST-EVENT SUMMARY EVENT SITE EPTHOUGHTLEADERS.COM EP_BIOMAN EXECUTIVE PLATFORMS HIGHLIGHTS 200+ Attendees 99% 85% Director-Level have personal budgetary 120+ and Above responsibilities over $5M GLOBAL ORGANIZATIONS 71% have an annual company revenue over $1B Keynotes, case studies, “Excellent ‘virtual’ roundtable discussions, conference with a broad 30+ workshops and more range of presentations and great information exchange on technologies transforming the biopharmaceutical industry.” 20+ Head, Global Biologics Solution Operating Unit providers Days of virtual networking, learning and 2 sharing ideas BIOMANWORLD.COM BMWS20 2 SPEAKERS Here are just a few of our speakers: Juan Andres Patrick Yang Chief Technical Operations Chairman at Acepodia and Quality Officer & Founding Board Director at Sana Biotechnology Lou Schmukler Michael Kamarck EVP and President, Global Product Chief Technology Officer Development & Supply Alison Moore Pam Cheng Chief Technology Officer EVP, President Global Operations & IT Jerry Cacia Derek Adams SVP, Head Global Technical Development Chief Technology and Manufacturing Officer John Pinion Brendan O’Callaghan EVP Translational Sciences, SVP & Global Head Chief Quality Operations Officer of Specialty Care IA Arleen Paulino Jens Vogel SVP Global Manufacturing SVP & Global Head of Biotech Charlene Banard Craig Kennedy Global Head of Technical Operations SVP Global Supply Chain Management for Cell & Gene Therapy BIOMANWORLD.COM BMWS20 3 ATTENDEES A2 Biotherapeutics Sr. Director, Tech Ops Bristol Myers Squibb Sr. Director Acadia Director QA Bristol Myers Squibb Sr. Research Associate Acceleron Pharma Inc VP, Quality Bristol Myers Squibb Director, Cell Culture Eng. CoE (Cell Therapy) Acepodia & Sana Biotechnology Chairman & Founding Board Director Bristol Myers Squibb Director of Manufacturing Adaptimmune Chief Patient Supply Officer Bristol Myers Squibb Engineer ADMA Biologics VP of Quality BMS - Cell Therapy Tech Ops Sr. Director of Development Operations Advantage Pharma Solutions Managing Principal BMS/Juno Therapeutics VP | Cell Therapy Product & Analytical Dev. Akron Biotechnology VP of Corporate Development Bristol-Myers Squibb Head, S12 CAR-T MSAT Akron Biotechnology VP of Product Development Director Alector VP, Quality CASI Pharmaceuticals Supply Management Alkermes Associate Director, Tech. Ops/Ext. Ops - Biologics Catalent VP, Biologics Product Development Allako VP Technical Operations Celldex Therapeutics Executive Director, Allogene Therapeutics Executive Director, Mfg Plant Manager Supply and Operations Management, MBA Allogene Therapeutics Chief Technology Officer Century Therapeutics Chief Technology Officer Allogene Therapeutics VP, Supply Chain and External Mfg Cidara Director, process chemistry Amgen VP Process Development Codexis AVP, Technical Operations Amgen VP, Attribute Sciences Codexis Director, Pharmaceutical Development/CMC Amgen SVP Global Manufacturing Denali Therapeutics CTMO Amgen VP Global Supply Chain Denali Therapeutics Head of Quality Assurance and Quality Control Amgen Executive Director DNAtrix, Inc. SVP, Manufacturing Amgen Director of Engineering Esperion Therapeutics VP, Manufacturing Planning & Operations Andelyn Biosciences VP, Head of Program Mgmt and Business Dev. FerGene SVP Technical Operations Arcutis Biotherapeutics SVP, Operations Flagship Pioneering Operating Partner Artisan Bio Chief Technology Officer Genentech Product Strategy Leader Artisan Bio VP, Cell Technologies & Entrepreneur in Residence Genentech Sr. Advisor, Cell and Gene Therapy AstraZeneca Head of Pharmaceutical Quality Systems Genentech Sr. Director & Head of Global Supplier Quality AstraZeneca EVP, President Global Operations & IT Genentech Global Head of Manufacturing Sciences Atara Bio Head of Product Quality Leadership and Technology, Drug Substance Atara Biotherapeutics Sr. Director, Strategic Planning & Operations Genentech Product Strategy Leader Autolus Sr. Director, Global Planning Genentech Head of Automation Autolus VP, Engineering and Supply Chain Genentech Head of Technical Development Leaders Bayer Director of Global Quality - Biotech Genentech VP General Manager Bayer SVP & Global Head of Biotech Genentech SVP, Head Global Technical Development Bayer Healthcare SUT Platform Engineer Gilead Sciences Executive Director of Quality Bayer Pharmaceuticals SVP & Global Head of Biotech Gilead Sciences Director Beam Therapeutics SVP, Technical Operations Gilead Sciences VP, Quality and Compliance Biogen Director, Product Development Quality Halozyme Therapeutics Director, Supply Chain BioMarin VP, Global Quality Operations Homology Medicines Sr. Director Downstream Process Development BioMarin Exec. Director, Strategic Network Planning Intellia Therapeutics SVP Technical Operations bluebird bio SVP, Quality Intergalactic Therapeutics SVP bluebird bio Chief Technology and Manufacturing Officer Just-Evotec Biotherapeutics VP Manufacturing bluebird bio SVP, Pharmaceutical Dev. & Technology Kite Pharma Director, GMSAT Bristol Myers Squibb Director Kite Pharma Exec Director, Quality, Corporate Quality Bristol-Myers Squibb VP Cell Therapy Mfg Science & Technology Kite Pharma VP Global Supply Chain Bristol-Myers Squibb EVP & President, Global Product Dev. & Supply Kite Pharma SVP, Quality and Compliance Bristol-Myers Squibb Exec. Director, Global Product Development Kyowa Kirin USA Holdings Global Head QA and Supply Business Insights and Analytics Merck & Co. SVP Global Supply Chain Management Bristol Myers Squibb Research Associate, Process Development Merck & Co. AVP Global Technical Operations Bristol Myers Squibb VP, Global Cell Therapy Manufacturing MilliporeSigma Site Director BIOMANWORLD.COM BMWS20 4 ATTENDEES Moderna Chief Technical Operations and Quality Officer Sanofi Genzyme VP and General Manager, Sanofi Allston Site Neurocrine Biosciences VP, Commercial Manufacturing & Supply Chain Sanofi Genzyme MSAT Head Notch Therapeutics Director, Manufacturing SaudiVax Managing Director Notch Therapeutics VP, Process Sciences Scilex Pharmacueticals Chief Technical Officer Novartis Global Head QA Compliance Sorrento Therapeutics Inc Sr. Director, Manufacturing Novartis Global Head of Technical Operations StealthCo Head, BD for Cell & Gene Therapy Takeda Head of Cell Bank Mfg and Center of Excellence Novartis Gene Therapies Sr Director, Site Quality Head Takeda SVP, Head Biologics Operating Unit Oncorus VP CMC Operations Takeda Pharmaceutical VP, Head of Engineering Biologics OpU Orchard Therapeutics VP Engineering and Facilities Takeda Pharmaceuticals Director Technical Operations Orchard Therapeutics Chief Technical Officer Tessa Therapeutics Ltd Chief Operating Officer PACT Pharma, Inc. SVP Manufacturing Thermo Fisher Scientific VP & General Manager, Biologics Pfenex Inc. Sr. Director - Commercial Strategy Tmunity Therapeutics Inc Chief Technology Officer Pfizer Sr. Director Turnstone Biologics, Inc. SVP, Technical Operations Prothelia Chief Technology Officer Ultragenyx VP Technical Development Regeneron SVP Preclinical Mfg & Process Development, Ultragenyx Sr. Director, Quality Assurance Research & Development Sciences Ultragenyx Gene Therapy VP, Quality Assurance REGENXBIO Inc. VP, Process Development Ultragenyx Pharmaceutical EVP Translational Sciences, Sana Biotechnology EVP Technical Operations Chief Quality Operations Officer Sangamo Sr. Director DP Formulation & Fill/Finish Vir Biotechnology Chief Technology Officer Sangamo Therapeutics VP, Supply Chain Management & Business Ops Xencor Sr. Director, CMC Operations Sanofi Production Unit Head - BioSurgery Yuan Xu Former CEO Sanofi VP Site Head of Framingham Biologics Ziopharm Oncology SVP Technical Operations Sanofi Head, Sanofi Manufacturing System / OpEx Ziopharm Oncology Sr. Director, Technical Operations CMC, Sanofi SVP & Global Head of Specialty Care IA Gene Therapy “I really enjoyed the BMWS format and that the presentations were recorded and available so fast after they were given. I missed some of the talks I wanted to hear but could go back and see the video recording of the section. Amazing speakers as always!” - Head of Quality Assurance and Quality Control BIOMANWORLD.COM BMWS20 5 VIDEOS Here are a few of the most popular presentations from BMWS20. COVID-19 MAbs at Pandemic Speed, BMWS20 – Opening Chairs’ Remarks and Keynote: Volumes and COGs Reinventing a Biopharma Company for the 21st Century Fighting COVID-19 with Innovation Accelerating Delivery of New Medicines – A Race Against Time Panel: Looking at the Big Picture: Where is Our Panel: The Past, Present and Future of the Industry Going, and How Should We Get There? Global Bio-Pharma Supply Chain BIOMANWORLD.COM BMWS20 6 INTERVIEW This video/podcast interview was recorded during BMWS20. Transport Simulation Testing of Your Therapy: A Better Approach Than “Real World” Shipping Tests In this interview, Gary Hutchinson of Modality Solutions shares his experience working with his clients to simulate how therapies can survive being transported from where they are made to where they are needed. Modality Solutions’ expertise in designing and understanding the supply chains underpinning the pharmaceutical industry is second to none, and Gary’s insights will help companies save time and avoid losing product as the world waits for delivery of life-saving therapies. “It is always very interesting to learn how leaders identify perceived constraints and turned them into manageable limitations. The COVID-19
Recommended publications
  • SANGAMO THERAPEUTICS, INC. (Exact Name of Registrant As Specified in Its Charter)
    UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended September 30, 2017 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission file number 000-30171 SANGAMO THERAPEUTICS, INC. (exact name of registrant as specified in its charter) Delaware 68-0359556 (State or other jurisdiction of (IRS Employer incorporation or organization) Identification No.) 501 Canal Blvd Richmond, California 94804 (Address of principal executive offices) (510) 970-6000 (Registrant’s telephone number, including area code) Indicate by check mark whether the registrant (1) has filed all reports required to be filed by section 13 or 15(d) of the Securities Act of 1934 during the preceding 12 months (or for such shorter period that the registrant was required to file such reports), and (2) has been subject to such filing requirements for the past 90 days. Yes ☒ No ☐ Indicate by check mark whether the registrant has submitted electronically and posted on its corporate Web site, if any, every Interactive Data File required to be submitted and posted pursuant to Rule 405 of Regulation S-T (§ 232.405 of this chapter) during the preceding 12 months (or for such shorter period that the registrant was required to submit and post such files). Yes ☒ No ☐ Indicate by check mark whether the registrant is a large accelerated filer, an accelerated filer, a non-accelerated filer, a smaller reporting company, or an emerging growth company.
    [Show full text]
  • NASDAQ Stock Market
    Nasdaq Stock Market Friday, December 28, 2018 Name Symbol Close 1st Constitution Bancorp FCCY 19.75 1st Source SRCE 40.25 2U TWOU 48.31 21st Century Fox Cl A FOXA 47.97 21st Century Fox Cl B FOX 47.62 21Vianet Group ADR VNET 8.63 51job ADR JOBS 61.7 111 ADR YI 6.05 360 Finance ADR QFIN 15.74 1347 Property Insurance Holdings PIH 4.05 1-800-FLOWERS.COM Cl A FLWS 11.92 AAON AAON 34.85 Abiomed ABMD 318.17 Acacia Communications ACIA 37.69 Acacia Research - Acacia ACTG 3 Technologies Acadia Healthcare ACHC 25.56 ACADIA Pharmaceuticals ACAD 15.65 Acceleron Pharma XLRN 44.13 Access National ANCX 21.31 Accuray ARAY 3.45 AcelRx Pharmaceuticals ACRX 2.34 Aceto ACET 0.82 Achaogen AKAO 1.31 Achillion Pharmaceuticals ACHN 1.48 AC Immune ACIU 9.78 ACI Worldwide ACIW 27.25 Aclaris Therapeutics ACRS 7.31 ACM Research Cl A ACMR 10.47 Acorda Therapeutics ACOR 14.98 Activision Blizzard ATVI 46.8 Adamas Pharmaceuticals ADMS 8.45 Adaptimmune Therapeutics ADR ADAP 5.15 Addus HomeCare ADUS 67.27 ADDvantage Technologies Group AEY 1.43 Adobe ADBE 223.13 Adtran ADTN 10.82 Aduro Biotech ADRO 2.65 Advanced Emissions Solutions ADES 10.07 Advanced Energy Industries AEIS 42.71 Advanced Micro Devices AMD 17.82 Advaxis ADXS 0.19 Adverum Biotechnologies ADVM 3.2 Aegion AEGN 16.24 Aeglea BioTherapeutics AGLE 7.67 Aemetis AMTX 0.57 Aerie Pharmaceuticals AERI 35.52 AeroVironment AVAV 67.57 Aevi Genomic Medicine GNMX 0.67 Affimed AFMD 3.11 Agile Therapeutics AGRX 0.61 Agilysys AGYS 14.59 Agios Pharmaceuticals AGIO 45.3 AGNC Investment AGNC 17.73 AgroFresh Solutions AGFS 3.85
    [Show full text]
  • ARCTURUS THERAPEUTICS HOLDINGS INC. (Exact Name of Registrant As Specified in Its Charter)
    UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 8-K CURRENT REPORT Pursuant to Section 13 or 15(d) of the Securities Exchange Act of 1934 Date of Report (Date of earliest event reported): May 20, 2020 ARCTURUS THERAPEUTICS HOLDINGS INC. (Exact name of registrant as specified in its charter) Delaware 001-38942 32-0595345 (State or other jurisdiction (Commission (I.R.S. Employer of incorporation) File Number) Identification No.) 10628 Science Center Drive, Suite 250 San Diego, California 92121 (Address of principal executive offices) Registrant’s telephone number, including area code: (858) 900-2660 (Former name or former address, if changed since last report) Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions: ☐ Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425) ☐ Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12) ☐ Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b)) ☐ Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c)) Securities registered pursuant to Section 12(b) of the Act: Trading Name of each exchange Title of each class Symbol(s) on which registered Common stock, par value $0.001 per share ARCT The NASDAQ Stock Market LLC Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (§230.405 of this chapter) or Rule 12b-2 of the Securities Exchange Act of 1934 (§240.12b-2 of this chapter).
    [Show full text]
  • Medicines in Development -- Cell Therapy and Gene Therapy
    Medicines in Development -- Cell Therapy and Gene Therapy Alzheimer's Disease Drug Name Sponsor Indication Development Phase allogeneic mesenchymal Stemedica Cell Technologies Alzheimer's disease Phase II stem cell therapy (itMSCs) San Diego, CA www.stemedica.com AstroStem Nature Cell Alzheimer's disease Phase I/II stem cell therapy Seoul, South Korea www.stemcellbio.com Arthritis/Musculoskeletal Disorders Drug Name Sponsor Indication Development Phase AdipoCell™ U.S. Stem Cell intervertebral disc degeneration Phase II adipose-derived autologous Sunrise, FL www.us-stemcell.com stem cell therapy CybroCell™ SpinalCyte intervertebral disc degeneration Phase I/II human dermal fibroblast-based Houston, TX www.spinalcyte.com cell therapy ECCO-50 Cytori Therapeutics osteoarthritis of the knee Phase II completed (adipose-derived autologous stem San Diego, CA www.cytori.com and regenerative cell therapy) Medicines in Development: Cell Therapy and Gene Therapy ǀ 2018 1 Arthritis/Musculoskeletal Disorders Drug Name Sponsor Indication Development Phase IDCT DiscGenics lumbar degenerative disc disease Phase I/II (allogeneic injectable discogenic Salt Lake City, UT www.discgenics.com cell therapy) JointStem Nature Cell osteoarthritis of the knee Phase II mesenchymal stem cell therapy Seoul, South Korea www.stemcellbio.com mesenchymal stem cell therapy Medipost osteoarthritis Phase I/II completed Seoul, South Korea www.medipost.com PLX-PAD (emiplacel) Pluristem Therapeutics muscle injury following arthroplasty Phase III (human placental stromal cell
    [Show full text]
  • Kite, a Gilead Company, and Sangamo Therapeutics Announce Collaboration to Develop Next-Generation Engineered Cell Therapies for the Treatment of Cancer
    Sangamo Therapeutics Logo Kite, a Gilead Company, and Sangamo Therapeutics Announce Collaboration to Develop Next-Generation Engineered Cell Therapies for the Treatment of Cancer February 22, 2018 -- Kite to Receive Exclusive License to Leverage Sangamo's Gene Editing Technology in Allogeneic and Autologous Cell Therapy Programs in Oncology -- SANTA MONICA, Calif. and RICHMOND, Calif., Feb. 22, 2018 /PRNewswire/ -- Kite, a Gilead Company (Nasdaq: GILD) and Sangamo Therapeutics, Inc. (Nasdaq: SGMO) today announced the companies have entered into a worldwide collaboration using Sangamo's zinc finger nuclease (ZFN) technology platform for the development of next-generation ex vivo cell therapies in oncology. Kite will use Sangamo's ZFN technology to modify genes to develop next-generation cell therapies for autologous and allogeneic use in treating different cancers. Allogeneic cell therapies from healthy donor cells or from renewable stem cells would provide a potential treatment option that can be accessed directly within the oncology infusion center, thus reducing the time to infusion for patients. Under the terms of the agreement, Sangamo will receive an upfront payment of $150 million and is eligible to receive up to $3.01 billion in potential payments, aggregated across 10 or more products utilizing Sangamo's technology, based on the achievement of certain research, development, regulatory and successful commercialization milestones. Sangamo would also receive tiered royalties on sales of potential future products resulting from the collaboration. Kite will be responsible for all development, manufacturing and commercialization of products under the collaboration, and will be responsible for agreed upon expenses incurred by Sangamo. "This collaboration between Kite and Sangamo brings together two leading platforms to develop best-in-class cell therapies in oncology," said Sandy Macrae, President and Chief Executive Officer of Sangamo.
    [Show full text]
  • ULTRAGENYX PHARMACEUTICAL INC. (Exact Name of Registrant As Specified in Its Charter)
    UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, DC 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934. For the quarterly period ended September 30, 2015 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934. For the transition period from to . Commission File No. 001-36276 ULTRAGENYX PHARMACEUTICAL INC. (Exact name of registrant as specified in its charter) Delaware 27-2546083 (State or other jurisdiction of (I.R.S. Employer incorporation or organization) Identification No.) 60 Leveroni Court, Novato, California 94949 (Address of principal executive offices) (Zip Code) (415) 483-8800 (Registrant’s telephone number, including area code) Indicate by check mark whether the registrant (1) has filed all reports required to be filed by Section 13 or 15(d) of the Securities and Exchange Act of 1934 during the preceding 12 months (or for such shorter period that the registrant was required to file such reports), and (2) has been subject to such filing requirements for the past 90 days. YES R NO ☐ Indicate by check mark whether the registrant has submitted electronically and posted on its corporate Website, if any, every Interactive Data File required to be submitted and posted pursuant to Rule 405 of Regulation S-T (§ 232.405 of this chapter) during the preceding 12 months (or for such shorter period that the registrant was required to submit and post such files). YES R NO ☐ Indicate by check mark whether the registrant is a large accelerated filer, an accelerated filer, a non-accelerated filer, or a smaller reporting company.
    [Show full text]
  • Year: 2017 Name: Amy Corneli Title: Associate Professor Address: 2200 West Main Street, Durham, NC 27705 Please Complete the Applicable Rows in the Following Table
    DCRI COMMERCIAL RELATIONSHIPS TRACKING FORM Year: 2017 Name: Amy Corneli Title: Associate Professor Address: 2200 West Main Street, Durham, NC 27705 Please complete the applicable rows in the following table. Monetary amounts are on an annual basis. Company: (1) A Research (2) A (3) Educational Activities or (4) Consulting or Other (5) Consulting or (6) I (7) I Have Grant or Research Lectures for this Company Services (Including Other non-CME Receive Equity in this Contract from Grant or Generates Revenue for CME) for this Company Services for this Significant Company this Company Contract from Duke Generates Personal Company Generates Personal Partially this Company Income Personal Income Royalties Supports My Supports My from this University Research <$5K $5K– <$5K $5K– >$25K <$5K $5K– >$25K Company <$5 >$5K/1% $25K $25K $25K Salary Projects >$25K K Abbott Laboratories Abbott Vascular Business (formerly Abbott Vascular Devices) AbbVie Abiomed Acceleron Pharma Inc Acerta Pharma ACIST Medical Systems, Inc AegisCN LLC Aires Pharmaceuticals Akili Interactive Labs Akros Pharmaceuticals page 1 of 7 Alexion Amgen, Inc Amylin Pharmaceuticals, Inc. ARCA biopharma Armis Biopharma Astellas Scientific and Medical Affairs Inc AstraZeneca Baseline Study LLC Basilea Pharmaceutica International Ltd Bayer AG Biosensors International Group Ltd Boehringer Ingleheim Boston Scientific Corporation Bristol Myers Squibb Cempra Pharmaceuticals, Inc. ContraFect Corporation CPC Clinical Research CryoLife Inc 2 of 7 CSL Limited DAIICHI SANKYO COMPANY, LIMITED Datavant Inc David H Murdock Inst for Business and Culture Duke Clinical Research Institute Durata Therapeutics Eiger BioPharmaceutical s Inc Einstein HC Network Philadelphia Elesvier Eli Lilly & Company Eureka Therapeutics Evalve, Inc. Ferring Pharmaceuticals Filament BioSolutions Galectin Therapeutic Genentech General Electric Company Genetic Alliance 3 of 7 Genzyme Corporation German AFNET Gilead GlaxoSmithKline plc Grifols HeartFlow Intarcia Therapeutics, Inc.
    [Show full text]
  • Sangamo Therapeutics Reports First Quarter 2019 Financial Results
    Sangamo Therapeutics Logo Sangamo Therapeutics Reports First Quarter 2019 Financial Results May 8, 2019 Conference Call and Webcast Scheduled for 5:00 p.m. Eastern Time BRISBANE, Calif., May 8, 2019 /PRNewswire/ -- Sangamo Therapeutics, Inc. (NASDAQ: SGMO), a genomic medicine company, today reported first quarter 2019 financial results and recent business highlights. "With recent encouraging clinical data in hemophilia A gene therapy and ex vivo gene-edited cell therapy for beta thalassemia, we are moving closer to achieving our vision for Sangamo as an integrated genomic medicine company," said Sandy Macrae, CEO of Sangamo. "Our capabilities in gene therapy, ex vivo gene-edited cell therapy, in vivo genome editing and gene regulation enable us to address genetic diseases with appropriate technologies. We are investing in a diverse pipeline of products where we believe this suite of proprietary technologies is clinically relevant, where the underlying biology is well-characterized, and where there is a defined high unmet medical need." Recent Highlights Clinical In partnership with Pfizer, announced phase 1/2 interim data for SB-525, a gene therapy candidate for the treatment of adults with hemophilia A, demonstrating dose-dependent increases in Factor VIII (FVIII) activity in eight patients, with the two patients treated with the 3e13 vg/kg dose reaching normal FVIII levels Initiated dose expansion of the 3e13 vg/kg dose cohort for the SB-525 hemophilia A program, based on Safety Monitoring Committee recommendations Announced early data
    [Show full text]
  • 2016 Medicines in Development for Rare Diseases a LIST of ORPHAN DRUGS in the PIPELINE
    2016 Medicines in Development for Rare Diseases A LIST OF ORPHAN DRUGS IN THE PIPELINE Autoimmune Diseases Product Name Sponsor Official FDA Designation* Development Status Actemra® Genentech treatment of systemic sclerosis Phase III tocilizumab South San Francisco, CA www.gene.com Adempas® Bayer HealthCare Pharmaceuticals treatment of systemic sclerosis Phase II riociguat Whippany, NJ www.pharma.bayer.com ARA 290 Araim Pharmaceuticals treatment of neuropathic pain in patients Phase II Tarrytown, NY with sarcoidosis www.ariampharma.com ARG201 arGentis Pharmaceuticals treatment of diffuse systemic sclerosis Phase II (type 1 native bovine skin Collierville, TN www.argentisrx.com collagen) BYM338 Novartis Pharmaceuticals treatment of inclusion body myositis Phase III (bimagrumab) East Hanover, NJ www.novartis.com CCX168 ChemoCentryx treatment of anti-neutrophil cytoplasmic Phase II (5a receptor antagonist) Mountain View, CA auto-antibodies associated vasculitides www.chemocentryx.com (granulomatosis with polyangitis or Wegener's granulomatosis), microscopic polyangitis, and Churg-Strauss syndrome * This designation is issued by the FDA's Office of Orphan Products Development while the drug is still in development. The designation makes the sponsor of the drug eligible for entitlements under the Orphan Drug Act of 1983. The entitlements include seven years of marketing exclusivity following FDA approval of the drug for the designated use. Medicines in Development: Rare Diseases | 2016 1 Autoimmune Diseases Product Name Sponsor Official FDA
    [Show full text]
  • Family Conference Dec
    33RD ANNUAL NATIONAL MPS SOCIETY FAMILY CONFERENCE DEC. 19–21, 2019 • ORLANDO, FL #mpsfamily2019 MISSION STATEMENT The National MPS Society exists to cure, support and advocate for MPS and ML. Contents MPS and ML Chair Letter 1 Mucopolysaccharidoses (MPS) and Mucolipidosis (ML) are genetic lysosomal storage diseases (LSD) caused by the body’s inability to Board of Directors/Staff 2 produce specific enzymes. General Information 3 MPS I MPS IV Hotel Layout 5 MPS I H Hurler MPS IV A Morquio A Schedule 6 MPS I S Scheie Enzyme / Galactose 6-sulfatase MPS IV B Morquio B Speakers 10 MPS I H-S Hurler-Scheie Enzyme / a-L-Iduronidase Enzyme / B-Galactosidase Attendees 30 MPS VI Thank You to Our Sponsors 36 MPS II MPS II Hunter MPS VI Maroteaux-Lamy Notes 38 Enzyme / Iduronate sulfatase Enzyme / (arylsulfatase B) N-Acetylgalac-tosamine 4-sulfatase MPS Jingle Bell Run/Walk 41 MPS III Upcoming Events 42 MPS VII MPS III A Sanfilippo A Enzyme / Heparan N-sulfatase MPS VII Sly Enzyme / B-Glucuronidase MPS III B Sanfilippo B Enzyme / a-N- Acetylglucosaminidase MPS IX MPS III C Sanfilippo C Enzyme / Hyaluronidase Enzyme / Acetyl CoA: a-glycosaminide acetyltransferase ML II/III MPS III D Sanfilippo D Enzyme / N-Acetylglucosamine ML II I-Cell 6-sulfatase ML III Psuedo-Hurler polydystrophy Enzyme / N-acetylglucosamine-1- phosphotransferase Chair Letter I am so excited to be at the National MPS Society’s 33rd Annual Family Conference at the most magical place on earth! Our family conference has a special place in my heart. I dearly treasure the time I spend with all of you and the entire MPS and ML community each year.
    [Show full text]
  • Active Labelers Run Date : Aug 28, 2018
    Active Labelers Run Date : Aug 28, 2018 Labeler ID Labeler Name Contract Begin Date Contract End Date 00002 ELI LILLY AND COMPANY 01/01/1991 01/01/3000 00003 E.R. SQUIBB & SONS, LLC. 01/01/1991 01/01/3000 00004 HOFFMANN-LA ROCHE 01/01/1991 01/01/3000 00006 MERCK & CO., INC. 01/01/1991 01/01/3000 00007 GLAXOSMITHKLINE 01/01/1991 01/01/3000 00008 WYETH LABORATORIES 01/01/1991 01/01/3000 00009 PFIZER, INC 01/01/1991 01/01/3000 00013 PFIZER, INC. 01/01/1991 01/01/3000 00014 PFIZER, INC 01/01/1991 01/01/3000 00015 MEAD JOHNSON AND COMPANY 01/01/1991 01/01/3000 00023 ALLERGAN INC 01/01/1991 01/01/3000 00024 SANOFI-AVENTIS, US LLC 01/01/1991 01/01/3000 00025 PFIZER, INC. 01/01/1991 01/01/3000 00026 BAYER HEALTHCARE LLC 01/01/1991 01/01/3000 00029 GLAXOSMITHKLINE 01/01/1991 01/01/3000 00032 ABBVIE INC. 01/01/1991 01/01/3000 00037 MEDA PHARMACEUTICALS, INC. 01/01/1991 01/01/3000 00039 SANOFI-AVENTIS, US LLC 01/01/1991 01/01/3000 00046 AYERST LABORATORIES 01/01/1991 01/01/3000 00049 PFIZER, INC 01/01/1991 01/01/3000 00051 ABBVIE INC 10/01/1997 01/01/3000 00052 ORGANON USA INC. 01/01/1991 01/01/3000 00053 CSL BEHRING LLC 01/01/1991 01/01/3000 00054 WEST-WARD PHARMACEUTICALS CORP. 01/01/1991 01/01/3000 00056 BRISTOL-MYERS SQUIBB PHARMA CO. 01/01/1991 01/01/3000 00062 ORTHO MCNEIL PHARMACEUTICALS 01/01/1991 01/01/3000 00064 HEALTHPOINT, LTD.
    [Show full text]
  • Rebateable Manufacturers
    Rebateable Labelers – July 2021 Manufacturers are responsible for updating their eligible drugs and pricing with CMS. Montana Healthcare Programs will not pay for an NDC not updated with CMS. Note: Some manufacturers on this list may have some NDCs that are covered and others that are not. Manufacturer ID Manufacturer Name 00002 ELI LILLY AND COMPANY 00003 E.R. SQUIBB & SONS, LLC. 00004 HOFFMANN-LA ROCHE 00006 MERCK & CO., INC. 00007 GLAXOSMITHKLINE 00008 WYETH PHARMACEUTICALS LLC, 00009 PHARMACIA AND UPJOHN COMPANY LLC 00013 PFIZER LABORATORIES DIV PFIZER INC 00015 MEAD JOHNSON AND COMPANY 00023 ALLERGAN INC 00024 SANOFI-AVENTIS, US LLC 00025 PFIZER LABORATORIES DIV PFIZER INC 00026 BAYER HEALTHCARE LLC 00032 ABBVIE INC. 00037 MEDA PHARMACEUTICALS, INC. 00039 SANOFI-AVENTIS, US LLC 00046 WYETH PHARMACEUTICALS INC. 00049 ROERIG 00051 ABBVIE INC 00052 ORGANON USA INC. 00053 CSL BEHRING L.L.C. 00054 HIKMA PHARMACEUTICAL USA, INC. 00056 BRISTOL-MYERS SQUIBB PHARMA CO. 00065 ALCON LABORATORIES, INC. 00068 AVENTIS PHARMACEUTICALS 00069 PFIZER LABORATORIES DIV PFIZER INC 00071 PARKE-DAVIS DIV OF PFIZER 00074 ABBVIE INC 00075 AVENTIS PHARMACEUTICALS, INC. 00078 NOVARTIS 00085 SCHERING CORPORATION 00087 BRISTOL-MYERS SQUIBB COMPANY 00088 AVENTIS PHARMACEUTICALS 00093 TEVA PHARMACEUTICALS USA, INC. 00095 BAUSCH HEALTH US, LLC Page 1 of 19 Manufacturer ID Manufacturer Name 00096 PERSON & COVEY, INC. 00113 L. PERRIGO COMPANY 00115 IMPAX GENERICS 00116 XTTRIUM LABORATORIES, INC. 00121 PHARMACEUTICAL ASSOCIATES, INC. 00131 UCB, INC. 00132 C B FLEET COMPANY INC 00143 HIKMA PHARMACEUTICAL USA, INC. 00145 STIEFEL LABORATORIES, INC, 00168 E FOUGERA AND CO. 00169 NOVO NORDISK, INC. 00172 TEVA PHARMACEUTICALS USA, INC 00173 GLAXOSMITHKLINE 00178 MISSION PHARMACAL COMPANY 00185 EON LABS, INC.
    [Show full text]