CMC STRATEGY FOR AAV GENE THERAPIES IN THE AGE OF RMAT DESIGNATION

摘要

The Regenerative Medicine and Advanced Therapy (RMAT) designation by the FDA allows for faster, and more streamlined approvals of cell and gene therapies for unmet medicinal needs. Advantages of RMAT designation include all of the benefits of the fast-track and breakthrough designation programs, including early interactions with the FDA. Voyager's VY-AADC program, an Adeno Associated Virus (AAV) gene therapy approach for advanced Parkinson's Disease (PD), was one of the therapies granted RMAT designation in 2018.