JAMA Network December 7, 2021
The FDA has partnered with the National Institutes of Health (NIH), 10 pharmaceutical companies, and 5 nonprofit organizations to speed the development of gene therapies for rare diseases—those that affect fewer than 200 000 people.
NIH Director Francis S. Collins, MD, PhD, explained the mission and name of the new collaboration—the Bespoke Gene Therapy Consortium (BGTC)—which is part of the NIH Accelerating Medicines Partnership program. “Most rare diseases are caused by a defect in a single gene that could potentially be targeted with a customized or ‘bespoke’ therapy that corrects or replaces the defective gene,” Collins said in a statement. More than 80% of the 7000 or so known rare diseases result from a single faulty gene, according to the NIH.
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