{"id":59338,"date":"2021-10-27T17:43:57","date_gmt":"2021-10-27T21:43:57","guid":{"rendered":"https:\/\/rarediseases.org\/nih-fda-and-15-private-organizations-join-forces-to-increase-effective-gene-therapies-for-rare-diseases\/"},"modified":"2021-10-27T17:43:57","modified_gmt":"2021-10-27T21:43:57","slug":"nih-fda-and-15-private-organizations-join-forces-to-increase-effective-gene-therapies-for-rare-diseases","status":"publish","type":"post","link":"https:\/\/rarediseases.org\/es\/nih-fda-and-15-private-organizations-join-forces-to-increase-effective-gene-therapies-for-rare-diseases\/","title":{"rendered":"NIH, FDA and 15 private organizations join forces to increase effective gene therapies for rare diseases"},"content":{"rendered":"

October 27, 2021<\/strong> – The National Institutes of Health, U.S. Food and Drug Administration, 10 pharmaceutical companies and five non-profit organizations 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\u00a0Bespoke Gene Therapy Consortium (BGTC)<\/a>, part of the\u00a0NIH Accelerating Medicines Partnership (AMP) program<\/a>\u00a0and 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.<\/p>\n

\u201cThe Bespoke Gene Therapy Consortium is a strong, collaborative initiative centered around patients and families. NORD is thrilled to be on the forefront of gene therapy development alongside this coalition, and we look forward to contributing to research and data collection that will truly make a difference for the nearly 30 million Americans living with a rare disorder.\u201d said Ed Neilan, M.D., Ph.D., Chief Scientific and Medical Officer, NORD<\/strong><\/p>\n

For nearly four decades, the National Organization for Rare Disorders (NORD) has made it a mission to explore and elevate care, cures and treatment for rare diseases \u2013 regardless of how many people a certain disease might affect. The BGTC is a resourceful and likeminded coalition of key stakeholders and it will help enhance the development of rare disease treatment and research for the next generation of patients.<\/p>\n

Private partners of the BGTC include:<\/p>\n