RARE Daily

Bespoke Gene Therapy Consortium Publishes Playbook

February 7, 2024

Rare Daily Staff

The Bespoke Gene Therapy Consortium has published its first playbook, which is intended to help accelerate the development of adeno-associated virus gene therapies for rare diseases.

The BGTC Regulatory Playbook provides a roadmap for streamlining product development and navigation of the regulatory pathway for AAV gene therapies, with the goal of getting new treatments to patients sooner. The playbook will be updated over time, and future versions will be published online.

Developed by the Accelerating Medicines Partnership and backed by the Foundation for the National Institutes of Health, the BGTC is a public-private partnership between the National Institutes of Health, the U.S. Food and Drug Administration, life science companies, and patient foundations.

“Gene therapies offer promise to thousands of patients with rare diseases, but progress is slowed by uncertainties about manufacturing and a clear regulatory path,” said Julie Gerberding, President and CEO of the FNIH. “The BGTC partnership brings experts across the biosciences ecosystem together to define the best ways to safely advance these treatments and encourage increased investments in their development.”

More than 30 million people in the United States live with the devastating effects of rare diseases. There are more than 10,000 rare diseases, 80 percent of which are caused by genetic defects. These individuals often lack access to effective treatment, as knowledge and research funding for many rare diseases often lag compared to more common diseases. With the current approach of targeting one rare disease at a time, there are no effective business models to return the investments needed to bring a single rare disease therapy for a small population to market.

The playbook was created as a “one-stop-shop guide” for clinical and drug development researchers who are working to bring AAV gene therapies to rare disease patients. It addresses the critical steps before the submission of Investigational New Drug applications for the first human studies and includes overviews of regulatory best practices as well as case studies of existing AAV gene therapies.

Future versions of the playbook will incorporate learnings from the BGTC’s efforts to streamline navigation of the regulatory pathway, including consortium members’ experience with proposals to minimize the amount of analytical testing and toxicology studies necessary for IND approval, without compromising patient safety.

BGTC expects the playbook will benefit future investigators as they develop novel AAV gene therapies and in the process change the definition of a disease of commercial interest.

Photo: Julie Gerberding, President and CEO of the FNIH

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