NIH and FDA Lead Coalition to Accelerate Gene Therapy Development for Rare Diseases

Published on: 

The National Institutes of Health (NIH), FDA, and fifteen private organizations are partnering together to form the Bespoke Gene Therapy Consortium.

The National Institutes of Health (NIH) announced the formation of the Bespoke Gene Therapy Consortium (BGTC) on Oct. 27th, 2021. In partnership with FDA, ten pharmaceutical companies, and five non-profits, this coalition is aimed at accelerating development of gene therapies for rare diseases.

According to a NIH press release, while there are over 7000 rare diseases (diseases which affect less than 200,000 people) that affect as many as 30 million Americans, only two heritable diseases have FDA-approved gene therapies. BGTC intends to donate approximately $76 million over the course of five years to support various projects aimed at providing treatments for these underserved populations.

BGTC plans to support between four and six clinical trials that are each focused on a different rare disease. To qualify, the condition must be a rare, single-gene disease with no gene therapies or commercial programs in development. It must also have substantial groundwork in place such that preclinical and clinical studies can be started quickly.

“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,” said Joni L. Rutter, acting director of NIH’s National Center for Advancing Translational Sciences, in the press release. “The BGTC aims to make it easier, faster and less expensive to pursue bespoke gene therapies in order to incentivize more companies to invest in this space and bring treatments to patients.”


“Most rare diseases are caused by a defect in a single gene that could potentially be targeted with a customized or ‘bespoke’ therapy that corrects or replaces the defective gene,” said NIH director Francis S. Collins in the press release. “There are now significant opportunities to improve the complex development process for gene therapies that would accelerate scientific progress and, most importantly, provide benefit to patients by increasing the number of effective gene therapies.”

Source: NIH