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Rare Genetic Disease Partnership

A new public-private partnership plans to speed up the development of gene therapies for rare genetic diseases, Stat News reports.

It adds that the US National Institutes of Health and the Food and Drug Administration are partnering with 10 pharmaceutical companies and five nonprofit groups to form the Bespoke Gene Therapy Consortium. About 30 million Americans have a rare genetic disease, the NIH notes.

"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 NIH director, tells Stat News.

The NIH, FDA, and the private partners are to contribute $76 million over five years to fund the consortium, the NIH says. It adds that the main focus of the BGTC will be to better understand the biology of the adeno-associated viral vector that is often used in gene therapies.