18 October 2023
Unlocking the Future of AAV-Based Gene Therapies
The promise of adeno-associated virus (AAV) vector-based gene therapies is undeniable. These therapies have already demonstrated potential in treating conditions like spinal muscular atrophy, retinal dystrophy, and hemophilia B, with the FDA approving three treatments as of early 2023. Yet, despite these successes, many promising therapies fail during clinical trials.
