The newly launched Bespoke Gene Therapy Consortium (BGTC) is a public-private partnership organized by the Foundation for NIH that aims to bring together academic, industry, and NIH partners to accelerate AAV-based gene therapies.
The BGTC is seeking information on rare diseases and disorders that could be candidates for AAV gene therapy trials conducted by the BGTC. They will select 4-6 monogenic conditions for pilot trials that will help standardize vector generation, harmonize manufacturing practices, and streamline regulatory pathways. Suggestions for candidate conditions are being collected with a Disease Nomination form which asks for details 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, and both adult and pediatric conditions will be considered; however, there should not be any known commercial programs developing a gene therapy for that condition.
Nominations are due February 18, 2022 by 11:59 pm EST to BGTC@fnih.org.
Please check out the NBSTRN tools and resources to help support your nomination package. NBSTRN can help assist in your nomination application. Please schedule a consultation at nbstrn@nbstrn.org
Nomination Form:
References:
Nature Reviews Drug Discovery about the BGTC https://www.nature.com/articles/d41573-021-00193-6
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