FDA, NIH Announce Bespoke Gene Therapy Consortium to Focus on Rare Diseases

The U.S. Food and Drug Administration (FDA) and the National Institutes of Health (NIH) announced this week they are partnering with 15 companies and organizations in an effort to "optimize and streamline" the creation of gene therapies for the millions of Americans battling rare diseases.

The collaboration will be called the Bespoke Gene Therapy Consortium (BGTC) and will be managed by the Foundation for the National Institutes of Health. The consortium will be considered part of the NIH's Accelerating Medicines Partnership program, according to a Wednesday news release from the FDA and NIH.

The FDA defines gene therapy as a process through which a gene that is causing or has the potential to cause a disease in humans is altered in a way that treats or cures the problem. According to the FDA, there are more than 7,000 different kinds of rare diseases in the country, some of which children can inherit through their genes.

Thus far, the FDA said only two kinds of rare diseases that can be inherited have gene therapy products that have received FDA approval. The agency described gene therapy as "a promising therapeutic touch," noting that scientists know how to identify the genes that cause many of the rare diseases with which Americans are diagnosed.

NIH Director Francis Collins said in the Wednesday release "most" of the rare diseases humans battle have been linked to one problematic gene "that could potentially be targeted with a customized or 'bespoke' therapy that corrects or replaces" it.

"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," Collins said.

The FDA said the consortium will strive to develop scientific understanding that could support further development of one kind of therapy strategy known as adeno-associated virus (AAV) gene therapy and look into ways "to streamline regulatory requirements and processes for the FDA approval of safe and effective gene therapies." Those efforts will be supported by an estimated $76 million contribution from the NIH and the consortium's 15 private partners over the next five years, the FDA said.

Joni Rutter, the National Center for Advancing Translational Sciences' acting director at the NIH, noted in the release the comparative scarcity of rare disease patients can make it difficult for scientific research into therapy options for those diseases to receive funding.

"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."

The list of private partners contributing to the consortium includes biotech companies like Pfizer and nonprofits like the American Society of Gene and Cell Therapy in Milwaukee, the FDA said.

The FDA defines a rare disease as one that impacts less than 200,000 Americans, a definition that the agency says derives from the 1983 Orphan Drug Act. Americans battle more than 7,000 kinds of rare diseases in the U.S., which collectively impact an estimated 30 million people, according to the FDA.