NIH, FDA and 15 private organizations join forces to increase effective gene therapies for rare diseases

The National Institutes of Health, U.S. Food and Drug Administration, 10 pharmaceutical companies and five non-profit organizations, including Penn spinout Spark Therapeutics, have partnered to accelerate development of gene therapies for the 30 million Americans who suffer from a rare disease.

While there are approximately 7,000 rare diseases, only two heritable diseases currently have FDA-approved gene therapies. The newly launched Bespoke Gene Therapy Consortium (BGTC), part of the NIH Accelerating Medicines Partnership (AMP) program and project-managed by the Foundation for the National Institutes of Health (FNIH), aims to optimize and streamline the gene therapy development process to help fill the unmet medical needs of people with rare diseases.

Read More on the NIH website

Skip to content