NIH, FDA Aim to Cut Cost Barriers to Rare Disease Therapies

Feb. 24, 2021, 9:20 PM

Drug companies pursuing gene therapies for rare diseases could have a new financial model that makes developing these cutting-edge, lifesaving treatments more sustainable.

Francis S. Collins, director of the National Institutes of Health, said Wednesday that the agency is looking at expanding an existing partnership with industry and nonprofits to focus on gene therapies for ultra rare diseases. These diseases may only affect a dozen or so people and therefore the commercial incentive to develop treatments isn’t there.

The new effort, potentially called the Bespoke Gene Therapy Consortium, could come online within the coming months and would focus on creating...

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