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NIH and FDA Lead Coalition to Accelerate Gene Therapy Development for Rare Diseases

October 29, 2021

The National Institutes of Health (NIH) announced the formation of the Bespoke Gene Therapy Consortium (BGTC) on Oct. 27th, 2021. In partnership with FDA, ten pharmaceutical companies, and five non-profits, this coalition is aimed at accelerating development of gene therapies for rare diseases.

According to a NIH press release, while there are over 7000 rare diseases (diseases which affect less than 200,000 people) that affect as many as 30 million Americans, only two heritable diseases have FDA-approved gene therapies. BGTC intends to donate approximately $76 million over the course of five years to support various projects aimed at providing treatments for these underserved populations.

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