Executive Director Passage Bio Philadelphia, Pennsylvania
Since 2015, the FDA has approved approximately 15 gene therapy products, bringing new hope to patients with severe diseases that previously had no treatment options. Among these, nine are based on adenovirus or adeno-associated virus (AAV) vectors, which have emerged as a leading platform for gene delivery. Despite the absence of evidence indicating environmental or transmission risks, viral DNA and shedding studies remain a regulatory recommendation and a critical component of clinical trial design.
This presentation will provide an overview of viral shedding profiles observed in approved gene therapy products across various routes of administration. Key topics will include regulatory guidelines for viral shedding study design, critical parameters of AAV shedding data reported in regulatory filings and product package inserts, and existing knowledge gaps in AAV shedding monitoring. Additionally, opportunities to enhance shedding assessments will be explored.
Learning Objectives:
Understand the regulatory recommendations for shedding study in gene therapy trials
Learn the emerging virus shedding profiles of the approved gene therapy products
Discuss current gaps in monitoring AAV shedding and opportunities for improving future shedding study design
