10 pharma companies working with FDA, NIH to find treatments for rare diseases

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 necessary to develop, test and bring individualized gene therapy treatments for a single disease to market," Joni Rutter, PhD, acting director of the NIH's National Center for Advancing Translational Sciences, said in the news release.  

The consortium aims to make it easier and less expensive for drugmakers to pursue treatments for rare diseases. 

NIH Director Francis Collins, MD, PhD, said that most rare diseases are caused by a defect in a single gene that could be targeted with a bespoke therapy that corrects or replaces the defective gene. Most rare inherited diseases stem from a specific gene mutation that is already known, meaning gene therapy is a promising treatment approach. 

The NIH and other members of the consortium will contribute about $76 million over five years to support the consortium's efforts. 

The drugmakers involved include Biogen, Johnson & Johnson, Novartis, Pfizer, REGENXBIO, Spark Therapeutics, Takeda Pharmaceutical, Taysha Gene Therapies, Thermo Fisher Scientific and Ultragenyx.

Read the full news release here

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