What’s Next for AAV Production and Gene Therapy: The ABC of AAV and the Impact of AAV Biology on Efficient Vector Production
In this webinar, Dr. R. Jude Samulski, one of the pioneers of AAV gene therapy, professor at UNC School of Medicine, and the co-founder and chief scientific officer of AskBio, offers his perspective following four decades of groundbreaking research to explore the biology of AAV production. He shares a comprehensive review from the early days of the field to the development of research-grade material, culminating in the first efforts of GMP production. He will also share a summary of the status of AAV trials, as well as offer his thoughts on key areas of innovation we can expect in the years ahead.
Additionally, Dr. Ramin Baghirzade, global head of commercial for gene therapy solutions at Charles River, explores a fit-for-purpose AAV manufacturing platform, nAAVigation®, that leverages 30+ years of biologics experience, proprietary cell lines and process expertise, and in-house plasmid production which, combined with 100% in-house analytics, accelerates timelines to GMP for therapeutic developers.
About the Presenters
Dr. Jude Samulski
Professor of Pharmacology, The University of North Carolina
Jude Samulski PhD is the Scientific Co-Founder and former CEO of Asklepios Biopharmaceutical (AskBio), which was acquired by Bayer in 2021 in a multi-billion-dollar deal. One of the pioneers of adeno-associated virus (AAV) gene therapy, Dr. Samulski is Professor of Pharmacology of The University of North Carolina (UNC) and served as Director of the UNC Gene Therapy Center for eight years, where he led the production of an FDA approved AAV clinical vector used to treat children with Canavan's disease. He is a co-founder and/or advisor to numerous biotech companies, including Merlin, Applied Genetic Technologies and Ceregene.
Samulski received his PhD in Medical Microbiology and Immunology from the University of Florida, during which time he demonstrated the first use of AAV as a viral vector. He completed post-doctoral training at Princeton University. Samulski has over 20 patents filed or issued in the area of AAV vectors. He has served as a gene therapy consultant to the FDA and a former member of the Recombinant DNA Advisory Committee (RAC). He is a recipient of the President's Distinguished Research Award from the University of Pittsburgh.
Dr. Ramin Baghirzade
Global Head Commercial, Gene Therapy CDMO Services, Charles River
nAAVigation® Accelerates Gene Therapy Programs
The proven platform provides AAV-based gene therapy developers with an expedited, cost-effective, and predictable pathway to GMP manufacturing, with the capability to reduce timelines by 55% translating to fewer than 8 months compared to traditional workflows.
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