BGTC Launches Clinical Trial Portfolio and Regulatory Playbook
There are more than 10,000 known rare genetic diseases, affecting more than 30 million Americans and their families, but there is typically little to no commercial interest in developing treatments for a disorder that may impact only a dozen or so patients, leaving many such patients with little or no hope for beneficial treatments.
Gene therapies hold promise for treating such patients, but progress is slowed by uncertainties about manufacturing and a clear regulatory path. The Bespoke Gene Therapy Consortium (BGTC) partnership, now in its second year, brings experts across the biosciences ecosystem together to define the best ways to safely advance these treatments and encourage increased investments in their development.
The BGTC is part of the Accelerating Medicines Partnership® (AMP®) program, which recently commemorated its first decade as an unprecedented public-private partnership building bridges to drug candidates and platforms for some of the world’s most pressing health concerns.
Pathway to Impact
- BGTC announced in 2023 the eight rare diseases that will comprise the program’s initial clinical trial portfolio, which will pioneer a novel approach to the development of adeno-associated virus (AAV) gene therapy for rare diseases. The goal is to demonstrate that manufacturing and testing standards can provide a streamlined approval pathway for first-in-human clinical trials.
- BGTC announced in early 2024 the publication of version 1.0 of its Regulatory Playbook, a “one-stop-shop guide” for researchers who are working to develop AAV gene therapies, describing best practices and guidelines for rapidly and safely advancing these promising new therapies.
“With this clinical trial portfolio, we can build the bridge that creates a standardized and publicly available roadmap for all AAV gene therapies to follow, with repeatable solutions for templates, regulatory files, and manufacturing processes.”
Courtney Silverthorn, PhD, Vice President, Strategic Alliances & Innovation, at the FNIH