The Bespoke Gene Therapy Consortium reissues new adeno-associated virus biology research opportunities

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BGTC Gene Therapy Symposium Flyer

The Accelerating Medicines Partnership® (AMP®) Bespoke Gene Therapy Consortium (BGTC) is hosting an informational webinar for the reissued adeno-associated virus (AAV) biology research opportunities on July 14, 2022.  

The Accelerating Medicines Partnership® (AMP®) Bespoke Gene Therapy Consortium (AMP BGTC)'s mission is to bring together public, private, and non-profit stakeholders to accelerate gene therapy development to target rare diseases. Given the modest size of populations impacted by individual rare diseases and the high cost of developing and producing gene therapies, drug developers are reluctant to pursue these indications. This creates a situation where potentially life-saving therapies are inaccessible to most patients.  

To address these unmet medical needs, the National Institutes of Health (NIH), U.S. Food and Drug Administration (FDA), and other stakeholders have partnered to create the BGTC, which is coordinated by the Foundation for the National Institutes of Health (FNIH). The BGTC focuses on the development of adeno-associated virus (AAV) gene delivery technology. A major goal of the program is to explore the fundamental biology of AAVs and leverage this knowledge to enhance the efficacy and production of therapeutics.  

The AMP BGTC has reissued new Request for Proposals (RFP) AAV biology research opportunities: 

  • AAV Biology Gene Expression RFP (2022-BGTC003): This RFP aims to advance our understanding of how AAV transduces tissues and cells of clinical importance and develop approaches to improve transduction efficiencies in such tissues and cells significantly. The proposals focus on critical mechanical steps involved in transduction in extra or intracellular events. This will enhance the therapeutic impact of AAV gene therapy in humans. 

  • AAV Biology Vector Production RFP (2022-BGTC004): This RFP aims to revolutionize our understanding and improve the production of high-quality and high-titer recombinant AAV (rAAV) vectors for gene therapy in humans. This can be achieved by dissecting the AAV viral life cycle and the molecular steps involved in generating recombinant AAV sectors. The goal is to improve the production of AAV gene therapy vectors.  

Proposals are due on August 28, 2022, at 11:59 pm ET. To aid the application process, the BGTC is hosting an informational webinar for these reissued RFPs on July 14, 2022, at 11 am - 12:30 pm ET. Learn more about the webinar and register here. Additional resources can be on the AMP BGTC website.