To support the newly launched Accelerating Medicines Partnership® Bespoke Gene Therapy Consortium (BGTC), 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.
Two Requests for Proposals (RFPs) have been announced. The BGTC is seeking high-throughput screens or other promising developments to optimize individual steps of the adeno-associated virus (AAV) vector generation and human gene expression pathways.
The BGTC Steering Committee also seeks information on rare diseases and disorders that could be candidates for AAV gene therapy trials conducted by the BGTC. A Disease Nomination form is available to obtain 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. Patient groups, clinicians, and researchers are strongly encouraged to collaborate on preparing a disease or disorder nomination. Selected nominations will be invited to submit a full RFP at a later date.
The FNIH will hold informational webinars about each of these opportunities, with details to follow on the BGTC website. RFP submissions and completed Disease Nomination forms are due by February 18, 2022, to BGTC@fnih.org.
Please direct questions about NIAMS-specific topics to Emily Carifi.