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

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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 advance innovative manufacturing of AAV vectors for gene therapy.

CBER anticipates that addressing the need for gene therapy products for diseases affecting very small populations will also lead to technical developments that will advance the entire field of gene therapy.

Additionally, this research is intended to support advances in regulatory science that allow for development of science and risk-based guidelines to facilitate faster adoption of these innovative therapies.

Some specific areas of research could include the following, but applications should clearly describe the potential impacts of the proposed enabling technology on readiness for broad implementation in the biological product industry, and/or regulatory evaluation:• 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 (one batch provides approximately 5-20 doses)