AAV Vector manufacturing for diseases affecting very small populations

The summary for the AAV Vector manufacturing for diseases affecting very small populations grant is detailed below. This summary states who is eligible for the grant, how much grant money will be awarded, current and past deadlines, Catalog of Federal Domestic Assistance (CFDA) numbers, and a sampling of similar government grants. Verify the accuracy of the data FederalGrants.com provides by visiting the webpage noted in the Link to Full Announcement section or by contacting the appropriate person listed as the Grant Announcement Contact. If any section is incomplete, please visit the website for the Food and Drug Administration, which is the U.S. government agency offering this grant.

AAV Vector manufacturing for diseases affecting very small populations: 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 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)