Industry Videos

Adeno-Associated Virus-Mediated Gene Therapy - Advances, Immune Challenges, and Research Innovations

At FDA Grand Rounds, Dr. Ronit Mazur of CBER reviewed advances and persistent immunological challenges in adeno-associated virus (AAV)–mediated gene therapy, outlining how AAV’s favorable safety and durability have driven a surge in FDA approvals since 2017. She summarized AAV biology and contrasted viral vector platforms, traced the regulatory trajectory from the first IND in 1995 to multiple recent BLAs, and highlighted clinical setbacks tied to immune responses. Dr. Mazur then detailed CBER laboratory research on capsid engineering, deimmunization strategies, sex-based immune differences, and new critical quality attributes (CQAs) that influence safety and immunogenicity. Her presentation emphasized translational tools and assays the FDA is developing to predict and mitigate immune reactions and to inform manufacturing and regulatory review.