The Bespoke Gene Therapy Consortium, managed by the Foundation for the National Institutes of Health (NIH), has selected eight rare diseases for its clinical trials program. The consortium aims to accelerate gene therapy development targeting rare diseases with few therapeutic options. The BGTC will develop protocols for first-in-human trials for Charcot-Marie-Tooth disease type 4J, congenital hereditary endothelial dystrophy, Morquio A syndrome, multiple sulfatase deficiency (MSD), NPHP5 retinal degeneration, propionic acidemia (PCCB), retinitis pigmentosa 45 and spastic paraplegia 50.
The goal of this public-private partnership is to establish minimum standards for drug manufacturing and preclinical testing to speed up regulatory processes for AAV-based gene therapies. With roughly $100 million in funding from commitments made by 33 organizations including biopharmaceutical companies and nonprofits as well as 11 NIH centers and the FDA, half of which will be provided by NIH, the consortium hopes to provide clear guidance on safety issues regarding future organizations that progress gene therapies into human clinical trials.