AAV Success Studies | Retinal Dystrophy | Spinal Muscular Atrophy
Wednesday, May 19, 2021
Recent AAV gene therapy product approvals have catalyzed the field. This new class of therapies has shown the potential to bring transformative benefit to patients. With dozens of AAV treatments in clinical studies, all eyes are on the field to gauge its disruptive impact.
The panel assesses the largest challenges of the first two products, the lessons learned for the broader CGT field, and the extent to which they serve as a precedent to broaden the AAV modality.
- Is AAV gene therapy restricted to genetically defined disorders, or will it be able to address common diseases in the near term?
- Lessons learned from these first-in-class approvals.
- Challenges to broaden this modality to similar indications.
- Reflections on safety signals in the clinical studies?
Q&A10:50 AM – 11:05 AM