October 29, 2021 -- The U.S. Food and Drug Administration, the National Institutes of Health (NIH), and other organizations are joining forces to increase the development of gene therapies for rare diseases.
The organizations have created the Bespoke Gene Therapy Consortium (BGTC), which is part of the NIH Accelerating Medicines Partnership program and the Foundation for the National Institutes of Health. The consortium aims to optimize and streamline the gene therapy development process to help fill the unmet medical needs of people with rare diseases.
Researchers involved with the consortium will focus on the biological and mechanistic steps involved in adeno-associated virus vector production, vector delivery of genes into human cells, and how therapeutic genes are activated in target cells.
The BGTC program will develop a standard set of analytic tests to apply to the manufacture of viral vectors made by consortium researchers. It will also explore ways to streamline regulatory requirements and processes.
The NIH and private partners will contribute approximately $76 million over five years to support BGTC-funded projects. This includes approximately $40 million from the participating NIH institutes and centers. The National Center for Advancing Translational Sciences will contribute approximately $8 million over five years.
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