What’s Next for AAV Production and Gene Therapy?

Adeno-associated viruses (AAVs) have become one of the most widely used gene delivery vectors in the rapidly maturing field of gene therapy, renowned for their flexibility and safety profile, with a strong and growing track record in the clinic.

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, will use his perspective from four decades of groundbreaking research to discuss the biology of AAV production, as well as the numerous steps required to generate clinical-grade AAV vectors. Samulski will provide 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 offering his thoughts on key areas of innovation we can expect in the years ahead.

The second speaker will be Dr. Ramin Baghirzade, global head of commercial for gene therapy solutions at Charles River Laboratories (CRL). CRL has developed 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. Combined with 100% in-house analytics capabilities, this platform accelerates timelines to GMP material. Along with nAAVigation™, CRL offers full end-to-end support of AAV product development, from research to GMP.

A live Q&A session followed the presentation, offering a chance to pose questions to our expert panelists.