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22Nd Annual Meeting Final Program Guide American Society of Gene & Cell Therapy 22nd Annual Meeting Final Program Guide Washington, D.C., USA Washington Hilton www.asgct.org #ASGCT19 ASGCT-0918-245 ONE-TIME DELIVERY. BENEFITS FOR A LIFETIME. Voyager’s mission is to develop and deliver life-changing therapies to people around the world living with severe neurological diseases. This begins with a disciplined commitment to discovery, research, development and manufacturing, and extends to our thoughtful engagement with (P342) (P342patients, healthcare providers, caregivers and advocacy organizations. ASGCT 2019 POSTER PRESENTATIONS ORAL PRESENTATIONS MON • APRIL 29, 5:00 PM Multiple novel engineered AAV capsids demonstrate MON • APRIL 29, 11:00 AM enhanced brain and spinal cord gene transfer after Cell Specific Transdction of a Vectorized Anti-Tau systemic administration in adult mice (P240) Session: Neurologic Diseases (Columbia Hall) Antibody Using IV Dosing of a Blood Brain Barrier Penetrant AAV Capsid in Mice Session: Tools, Delivery and Neuro Capsids MON • APRIL 29, 5:00 PM (Monroe Room) Stability of rAAV Vectors: Response to Various Biochemical and Biophysical Stresses (P342) MON • APRIL 29, 11:30 AM Session: Vector and Cell Engineering, Production or Manufacturing (Columbia Hall) Intraparenchymal Spinal Cord Delivery of AAV Gene Therapy Provides Robust SOD1 Knockdown in Large TUE APRIL 30, 5:00 PM Mammal Spinal Cord for the Treatment of SOD1-ALS • Session: Tools, Delivery and Neuro Capsids Variability analysis of qPCR, ddPCR and potency assays (Monroe Room) for AAV vectors: implications for future development (P448) Session: RNA Virus Vectors (Columbia Hall) MON APRIL 29, 11:45 AM • Targeted in vivo Biopanning of AAV Capsid Libraries WED • MAY 1, 5:00 PM Using Cell Type-Specific RNA Expression Development of a High Cell Density Perfusion method for Session: Directed Evolution of AAV Vectors I Baculovirus Infected Insect Cells (BIICs) manufacturing (P726) (Georgetown Room) Session: AAV Vectors III (Columbia Hall) MON • APRIL 29, 11:45 AM WED • MAY 1, 5:00 PM Significant reduction of huntingtin gene expression in Characterization of AAV Percentage of Full Capsids cortex, putamen and caudate of large mammals with and Comparability Across Platforms (P743) combined putamen and thalamus infusions of VY-HTT01, Session: AAV Vectors III (Columbia Hall) an AAV gene therapy targeting huntingtin for the treatment of Huntington’s disease WED • MAY 1, 5:00 PM Session: Gene Silencing Approaches Optimization and Evaluation of two Potency Assays (IBR West Room) for AAV based Gene Silencing programs (P745) Session: AAV Vectors III (Columbia Hall) TUE • APRIL 30, 5:00 PM Evaluation of Tropism and Transduction Efficiency WED • MAY 1, 5:00 PM of AAV Variants in the CNS of NHP Using DNA/RNA Viral Clearance for rAAV Products in a Sf9/Baculovirus Barcode-seq Technology Manufacturing Process (P899) Session: Rational Engineering of AAV Vectors II Session: Vector and Cell Engineering, (Georgetown Room) Production or Manufacturing II (Columbia Hall) www.voyagertherapeutics.com � @VOYAGERTX TABLE OF CONTENTS A Welcome from Michele Calos, PhD ...........................................................2 Mobile App Instructions ................................................................................3 General Meeting Information .......................................................................4 Hotel Floor Plans .......................................................................................10 Program Committee & Abstract Planning Committee .................................15 Abstract Reviewers ....................................................................................16 Faculty List .................................................................................................20 Awards .......................................................................................................32 Schedule at a Glance ..................................................................................33 Program Schedule .......................................................................................34 Annual Meeting Supporters ......................................................................113 Annual Meeting Exhibitors ........................................................................114 Exhibit Hall Floor Plan ...............................................................................133 FINAL PROGRAM GUIDE • WASHINGTON, D.C. • 2019 1 A WELCOME FROM MICHELE CALOS, PHD A WELCOME FROM MICHELE CALOS, PHD Dear Colleagues, On behalf of the American Society of Gene & Cell Therapy (ASGCT), it is my pleasure to welcome you to the ASGCT 22nd Annual Meeting in Washington, DC. I am honored to join you and thousands of our colleagues from around the globe who share in our Society’s mission of advancing knowledge, awareness, and education to advance the discovery and clinical application of gene and cell therapies for alleviation of human disease. This scientific and educational program reflects the wonderful advancements that have occurred in gene and cell therapy over the past year. Our invited faculty includes nearly 150 investigators in our field, presenting cutting edge research in the 23 Scientific Symposia and overviews of important concepts and technologies in the seven Education Sessions. In addition, join me in welcoming former ASGCT President and Director of the Center for Cell Engineering at Memorial Sloan-Kettering Cancer Center, Dr. Michel Sadelain, who will present the George Stamatoyannopoulos Lecture on Tuesday. On Wednesday morning, the presentation of the Outstanding Achievement Award to Dr. John Rossi will occur followed by Dr. George Church on Wednesday afternoon presenting the Presidential lecture. We are also thrilled to share 1,000 abstracts featuring groundbreaking scientific and clinical advances across the breadth of our field that will be presented as oral presentations or at poster sessions. I look forward to the Annual Meeting program with an immense sense of excitement and pride in our field. On Wednesday evening, I am delighted to invite you to join the ASGCT leadership and me in celebrating the Society in our closing night reception. The reception takes place in the National Portrait Gallery of the Smithsonian Institute, where we will enjoy live music and dancing as well full access to the museum’s collections including the Orchids, American Presidents, and Henrietta Lacks exhibitions. Be sure to visit our Exhibit Hall to find out about the products and services offered by more than 100 partner companies, via conversations at their booths and more formal presentations in the Tools & Technologies Forum. Finally, I would like to thank everyone involved in the planning and execution of our Annual Meeting, especially the Program Committee, Scientific and Education Committees, abstract reviewers, staff and all of our volunteers. Their sustained support, dedication and hard work throughout the year now comes to fruition in what is sure to be a spectacular meeting. Lastly, thank you for attending our Society's Annual Meeting. I sincerely hope you enjoy the science, technology, networking, and camaraderie that we have built together! Best regards, Michele Calos, PhD President, ASGCT 2 FINAL PROGRAM GUIDE • WASHINGTON, D.C. • 2019 MOBILE APP INSTRUCTIONS DOWNLOAD THE ASGCT APP SEARCH "ASGCT" ON GOOGLE PLAY OR THE APPLE APP STORE Navigate the 22nd Annual Meeting like a pro with all-new ASGCT mobile app, powered by TripBuilder Media: Build your schedule and bookmark exhibitors Read all abstracts presented at the Annual Meeting Receive up-to-the-minute updates, including room and schedule changes Optimize your visit with local recommendations App support: Sponsored by: [email protected] STAY CONNECTED ON ASGCT WI-FI SAVE MOBILE DATA BY LOGGING ON THROUGH COMPLIMENTARY ANNUAL MEETING WI-FI NETWORK: ASGCT2019 PASSWORD: annualmeeting Sponsored by: FINAL PROGRAM GUIDE • WASHINGTON, D.C. • 2019 3 GENERAL MEETING INFORMATION GENERAL MEETING INFORMATION AMERICAN SOCIETY OF GENE & CELL THERAPY MISSION AND VISION The mission of ASGCT to advance knowledge, awareness, and education leading to the discovery and clinical application of genetic and cellular therapies to alleviate human disease. ASGCT’s strategic vision is to be a catalyst for bringing together scientists, physicians, patient advocates, and other stakeholders to transform the practice of medicine by incorporating the use of genetic and cellular therapies to control and cure human disease. ABSTRACT VOLUME - MOLECULAR THERAPY All abstracts accepted for presentation at the ASGCT 22nd Annual Meeting have been published in the May supplement of Molecular Therapy. Attendees are able to access a copy of the supplement online at the Molecular Therapy website, or have access to the abstracts through the Mobile App. ADMISSION Official name badges will be required for admission to all ASGCT sessions. All Annual Meeting attendees receive a name badge with their registration bag. Name badges should be worn at all times inside the Hilton, as badges will be used to control access to sessions and activities. Attendees are cautioned against wearing their name badges while away from the hotel as badges may draw unwanted attention to your status as visitors to Washington, DC. BUSINESS CENTER (FEDEX OFFICE) The Business Center (FedEx Office) is located in the Washington Hilton. It is open Monday – Friday from 7:00 am to 7:00 pm and on Saturday from 9:00 am – 3:00 pm. The Business Center is closed
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