What is the BGTC?
Launched in October 2021, the BGTC is the first initiative of the Accelerating Medicines Partnerships® (AMP®) program with a mission-driven focus for rare diseases.
The AMP® is a public-private partnership among the NIH, the U.S. Food and Drug Administration (FDA), multiple pharmaceutical and life sciences companies, nonprofits, and other organizations. Coordinated by the FNIH, AMP BGTC brings together partners from across the healthcare ecosystem to foster development of gene therapies for rare diseases, affecting populations too small for viable commercial development.
Why BGTC?
There are over 10,000 rare diseases that are caused by genetic defects with 25-30 million people in the United States living with the devastating effects of these rare diseases. These patients often lack access to effective treatment, as knowledge and research funding for many rare diseases often lags compared to more prevalent 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. [1]
A gene therapy frequently treats a disease by replacing the malfunctioning gene responsible for the condition with a “working version” of the gene by using a delivery system often called a “vector.” AAV gene therapies have been successfully used as interventions to treat genetic disorders and have received U.S. Food and Drug Administration (FDA) approval for human use.
The good news is that these gene therapies can be tailor-made for a very small population or even a single individual. However, the development process for these “bespoke” therapies is complex, expensive, and hampered by a lack of common biologic, manufacturing, and regulatory standards.
This is where the BGTC comes in!
The BGTC builds on the successful AMP model and aims to streamline the entire regulatory process and make gene therapies for rare diseases more available to patients who need them. The solution involves two areas of focus, as shown below:
Exploring AAV basic biology and translational implications
Advancing access to AAV technologies & vectors for bespoke clinical applications
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By leveraging up to eight clinical trial test cases and the unparalleled combined expertise of the consortium partners, we have developed a clinical development manual or “playbook” for developing gene therapies for rare diseases.
The BGTC Regulatory Playbook highlights the second objective, advancing access to AAV technologies, acting as a standard operational guide with the tools to embark on the regulatory process. In future versions of the playbook, the BGTC plans to decode efficiencies that streamline development and provide standards such as minimum regulatory requirements derived from our work to enable future investigations with novel AAV gene therapies for various genetic disorders. We define minimum regulatory requirements as regulatory activities that fulfill the essential safety and efficacy requirements outlined in the relevant sections of the Code of Federal Regulations. This may be distinct from the standard set of regulatory activities that commercial sponsors follow and that may be well known in the “industry”. Such activities may be better suited for commercial objectives and may not be feasible for noncommercial or academic rare disease sponsors who are the intended audience of this playbook.