Foundation for the National Institutes of Health (FNIH) Announces AMP BGTC’s Selection of Eight Rare Diseases for Clinical Trials, Paving the Way for Streamlined Gene Therapy Approvals

Foundation for the National Institutes of Health (FNIH) Announces AMP BGTC’s Selection of Eight Rare Diseases for Clinical Trials, Paving the Way for Streamlined Gene Therapy Approvals

Exciting news from the Foundation for the National Institutes of Health (FNIH) as the Accelerating Medicines Partnership® Bespoke Gene Therapy Consortium (AMP® BGTC) selects eight rare diseases for its clinical trial portfolio. This innovative approach to gene therapy development for rare diseases demonstrates the potential for streamlined approval pathways through manufacturing and testing standards. By establishing minimum standards and providing information to the public at no cost, the BGTC aims to expedite the delivery of customized gene therapies. This milestone enables further options for treating patients with rare genetic diseases and helps prepare the way for approved therapies. We congratulate the FNIH and the AMP BGTC for their commitment to advancing medical research and improving outcomes for patients.