Eight Rare Diseases, Including 4 Neurologic Conditions, Chosen for Accelerating Medicines Partnership® Bespoke Gene Therapy Consortium
The Accelerating Medicines Partnership® Bespoke Gene Therapy Consortium (AMP®BGTC) of the Foundation of the National Institutes of Health (FNIH) has chosen 8 rare diseases for its clinical trial portfolio, including 4 neurologic conditions:
- Charcot-Marie-Tooth disease type 4J
- Multiple Sulfatase Deficiency
- Propionic Acidemia (PCCB)
- Spastic paraplegia 50
along with other conditions
- Congenital Hereditary Endothelial Dystrophy
- Morquio A Syndrome
- NPHP5 Retinal Degeneration
- Retinitis pigmentosa 45
The goal of the consortium is to streamline the regulatory approval process for the development of gene therapies for rare diseases by establishing standard manufacturing and testing standards.
The AMP®BGTC represents a public-private partnership between public groups such as the National Institutes of Health and the Food and Drug Administration and private biopharmaceutical and life science companies, nonprofits, and other organizations. The announcement about these selections was issued during the American Society of Gene & Cell Therapy (ASGCT) 26th Annual Meeting in Los Angeles, CA, May 16-20, 2023. Selections were made by a panel of gene therapy experts following a scientific and technical review and using BGTC criteria, including lack of available treatments, an established disease model, and adequate proofs of concept and natural history data among other factors.
“With this clinical trial portfolio, we can build the bridge that creates a standardized and publicly available roadmap for all adeno-associated virus (AAV) gene therapies to follow, with repeatable solutions for templates, regulatory files, and manufacturing processes,” said Courtney Silverthorn, PhD, Associate Vice President, Science Partnerships, FNIH. “A single rare disease can affect hundreds of thousands of people globally, or merely a few dozen, and the BGTC aspires to make gene therapies more accessible and sustainable no matter the size of the patient population.”