FDA analysis of 30 years of AAV gene therapy submissions highlights common regulatory hurdles

Researchers affiliated with the US Food and Drug Administration reviewed investigational new drug applications submitted to the Center for Biologics Evaluation and Research spanning three decades from 1995 to 2025. The evaluation outlines the expansion of adeno-associated virus development over thirty years and highlights recurring factors that trigger regulatory holds during the review process. The authors note that the opinions and perspectives presented reflect the individual authors and do not necessarily represent official agency policies, while offering retrospective insights designed to guide successful product development.