July 12, 2022 – The Accelerating Medicines Partnership® Bespoke Gene Therapy Consortium (AMP® BGTC) is pleased to announce the completion of the first step of the disease selection process. The BGTC expert review team selected 14 candidate diseases out of the 63 nominations received. These selections were based upon the established required and preferred criteria, including but not limited to, the adequacy of the gene for insertion into an AAV vector, evidence of clinical benefit in gene therapy, and feasibility of conducting a clinical trial. A detailed description of the selection criteria and review process that was followed is available through this link.

Clinical trial proposals directed to one of the 14 bespoke indications can be submitted in response to the BGTC’s clinical trials RFP for final consideration for the BGTC’s clinical program. Applications are due by 11:59pm ET October 31, 2022 and an informational webinar will be held on August 30 from 11:30 am to 1:00 pm ET. We anticipate completing the final selection process in February 2023 and selecting 5-6 diseases to move forward with clinical trials. BGTC highly encourages collaboration between Investigators working in these disease areas, including collaboration with NIH Intramural Investigators, and engagement with relevant patient groups in the application process.

Through the BGTC’s partnership with the California Institute of Regenerative Medicine (CIRM), investigators working in California or collaborating with California-based research institutions and trial sites are eligible to apply for co-funding through CIRM’s existing funding opportunities. CIRM’s review process will coincide with the BGTC’s review process.

We would like to thank everyone who submitted disease nominations and the scientists who volunteered their time to serve on the review committee. We hope that the research and streamlined regulatory process generated by our clinical program will pave the way for clinical trials for many more rare diseases.

For more information about the project click here.

About AMP: AMP BGTC is one of many AMP programs expediting discovery around Alzheimer’s disease, Parkinson’s disease, Schizophrenia, Rheumatoid Arthritis and Lupus, Type II Diabetes, Common Metabolic Diseases, and Autoimmune and Immune-Mediated Diseases, all coordinated by the FNIH since the 2014 launch of the large-scale initiative. The AMP partnerships use cutting-edge scientific approaches to bring new medicines to patients by enhancing validation of novel, clinically relevant therapeutic targets and biomarkers. To learn more about AMP, visit https://fnih.org/AMP.

About the Foundation for the National Institutes of Health: The Foundation for the National Institutes of Health (FNIH) creates and manages alliances with public and private institutions in support of the mission of the NIH. The FNIH works with its partners to accelerate biomedical research and strategies against diseases and health concerns in the United States and across the globe. Established by Congress in 1990, the FNIH is a not-for-profit 501(c)(3) charitable organization. For additional information about the FNIH, please visit https://fnih.org.

ACCELERATING MEDICINES PARTNERSHIP and AMP are registered service marks of the U.S. Department of Health and Human Services.