NIH Foundation Expands Gene Therapy Clinical Trial Portfolio to Include Eight Rare Diseases.
The Accelerating Medicines Partnership Bespoke Gene Therapy Consortium (AMP BGTC) has selected eight rare diseases for its clinical trial portfolio, pioneering a novel approach to gene therapy development for rare diseases. Managed by the Foundation for the National Institutes of Health, AMP BGTC is a public-private partnership between the National Institutes of Health (NIH), the U.S. Food and Drug Administration (FDA), biopharmaceutical and life science companies, and nonprofit and other organizations to help speed the development and delivery of customized or ”bespoke” gene therapies that could treat the millions of people affected by rare diseases.