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2021-10-28| Policy

Big Pharma and Non-Profits Join NIH, FDA in New Rare Diseases Drive

by Joy Lin
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The US National Institutes of Health (NIH) and FDA have joined forces with 10 pharmas and five non-profits to boost the number of approved gene therapies for rare diseases.

The new partnership, called the Bespoke Gene Therapy Consortium (BGTC), will be part of the NIH Accelerating Medicines Program (AMP) and managed by the Foundation for the National Institutes of Health (FNIH). The NIH and private partners will contribute around $76 million in funding for the program over a five-year period. The participating NIH institutes and centers are expected to chip in over half the funds, at $39.5 million.

“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,” said NIH Director Francis S. Collins, M.D., Ph.D.

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