Public-Private Alliance Chooses Eight Rare Diseases for Focused Gene Therapy Development

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 h..