The National Institutes of Health has recently launched an Accelerating Medicines Partnership® Bespoke Gene Therapy Consortium (BGTC). This project will create tools to streamline the gene therapy development process, aiming to reduce associated costs and encourage companies to pursue gene therapies for rare genetic diseases.
In support of this endeavor, the Foundation for the National Institutes of Health (FNIH) has released multiple opportunities for researchers, clinicians, and patient advocates to contribute to the work of the consortium.
The BGTC is seeking proposals to advance the understanding of adeno-associated virus (AAV) biology and to develop solutions to optimize individual steps of AAV vector production and human gene expression pathways.
The following two Requests for Proposals (RFPs) have been announced, due August 28, 2022:
The BGTC Steering Committee also seeks nominations for rare diseases and disorders that could be candidates for AAV gene therapy trials conducted by the BGTC. Patient groups, clinicians, and researchers are strongly encouraged to collaborate on preparing a disease or disorder nomination.
Provide information about the rare disease and the patient population, prior R&D work, and high-level factors that would be considered in designing a clinical trial. Selected nominations will be invited to submit a full RFP at a later date.