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

The FDA, NIH, 10 pharmaceutical companies, and 5 nonprofit organizations have partnered to accelerate development of gene therapies for the 30 million Americans with rare diseases. Of approximately 7,000 rare diseases, only 2 currently have FDA-approved gene therapies. The newly launched Bespoke Gene Therapy Consortium, part of the NIH Accelerating Medicines Partnership program and project-managed by the Foundation for the National Institutes of Health, aims to optimize and streamline the gene therapy development process to help fill the unmet medical needs of people with rare diseases. Click here to read the full press release.