CBER seeks to advance the development of gene therapies for diseases affecting very small populations, potentially even single individuals, by enhancing innovations in the manufacture of Adeno-associated virus (AAV) vectors. Such innovations include the definition of critical quality attributes of AAV as vectors for gene therapy, development of advanced process analytics, development of a of non-proprietary AAV vectors, and demonstration of low-cost, non-proprietary GMP manufacturing methods for AAV vectors.This research will adv...