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Oct. 27, 2021

TOPIC: Press Releases, Research

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

Posted by Rohan Narayanan

October 27, 2021 – 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 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.

“The 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.” said Ed Neilan, M.D., Ph.D., Chief Scientific and Medical Officer, NORD

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 – 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.

Private partners of the BGTC include:

  • Biogen Inc., Cambridge, Massachusetts;
  • Janssen Research & Development, LLC, Raritan, New Jersey;
  • Novartis Institutes for BioMedical Research, Cambridge, Massachusetts;
  • Pfizer Inc., New York, New York;
  • REGENXBIO Inc., Rockville, Maryland.;
  • Spark Therapeutics, Philadelphia, Pennsylvania;
  • Takeda Pharmaceutical Company Limited, Deerfield, Illinois;
  • Taysha Gene Therapies, Dallas, Texas;
  • Thermo Fisher Scientific Inc., Waltham, Massachusetts; and
  • Ultragenyx Pharmaceutical, Novato, California.

The non-profit partners involved include:

  • The Alliance for Regenerative Medicine (ARM), Washington, D.C.;
  • The American Society of Gene and Cell Therapy, Milwaukee, Wisconsin;
  • CureDuchenne, Newport Beach, California;
  • National Organization for Rare Disorders (NORD), Quincy, Massachusetts; and
  • The National Institute for Innovation in Manufacturing Biopharmaceuticals (NIIMBL), Newark, Delaware.

For more information on the BGTC program, visit the NIH website.