Becker's Healthcare October 27, 2021
Ten drugmakers have partnered with the FDA and National Institutes of Health to form a consortium focused on finding treatments for rare diseases, the FDA announced Oct. 27.
The Bespoke Gene Therapy Consortium, which also includes five nonprofits, aims to accelerate research and development of gene therapy drugs that can treat the roughly 30 million Americans who suffer from a rare disease.
While there are about 7,000 known rare diseases, only two have FDA-approved gene therapies, the FDA said in a news release.
“Rare diseases affect 25 to 30 million Americans, but because any given rare disorder affects so few patients, companies often are reluctant or unable to invest the years of research and millions of dollars...
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