AAV Vector manufacturing for diseases affecting very small populations

The Food and Drug Administration (FDA) is seeking to advance the development of gene therapies for diseases affecting very small populations by enhancing innovations in the manufacture of Adeno-associated virus (AAV) vectors. This research aims to support advances in regulatory science and facilitate faster adoption of innovative therapies. The specific areas of research include definition of critical quality attributes of AAV vectors, development of advanced analytics, development of non-proprietary AAV vectors, and demonstration of low-cost, non-proprietary GMP manufacturing methods. Some key aspects of this opportunity include: • Definition of critical quality attributes of AAV vectors for gene therapy • Development of advanced analytics to enhance AAV vector manufacturing process knowledge and monitoring of critical quality attributes • Development of a non-proprietary suite of AAV vectors suitable for gene therapy applications for diseases affecting very small populations • Demonstration of the feasibility of low-cost, non-proprietary GMP manufacturing methods for AAV vectors at a scale appropriate for very small populations