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The National Institutes of Health, U.S. Food Drug Administration, and the
Foundation for the Institutes of Health unveiled a public-private partnership Wednesday aimed at accelerating gene therapies for the roughly 30 million Americans living with a rare genetic disease.

“They’ve waited a long time for something to be focused this way to address the incredibly wrenching stories we see all around us of children and adults with rare diseases where we can do a diagnostic test to tell them what they have but beyond that haven’t had much to offer,” Francis Collins, the pioneering genetics researcher and longtime NIH director, told STAT.


With $76 million over five years, the Bespoke Gene Therapy Consortium, or BGTC, will fund research into improving manufacturing processes and standardizing methods for preclinical testing of gene therapies. The initiative, which will be managed by the Foundation for the National Institutes of Health, includes 10 pharmaceutical companies and five nonprofit organizations, will also support four to six clinical trials, each focused on a different rare disease.

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