- The National Institutes of Health and Food and Drug Administration are banding together with drugmakers and nonprofits to speed the development of gene therapies for rare diseases.
- The group is launching the Bespoke Gene Therapy Consortium, with planned funding of $76 million over five years. About $40 million of that total will come from centers and institutes within the NIH, according to a statement.
- Industry partners for the effort span from major drugmakers such as Johnson & Johnson and Pfizer to gene therapy specialists Spark Therapeutics and Regenxbio. Thermo Fisher Scientific, a contract and development manufacturing organization that’s increasingly investing in gene therapy work, is also participating.
Gene therapies have the potential to offer a literal lifeline to people with rare diseases, many of which have no effective treatments. While each rare disease only affects a small number of patients, scientists have identified about 7,000 of them.
But despite considerable progress in gene therapy research over the past few decades, only two rare disease treatments — one for an inherited form of blindness, another for the neuromuscular disorder spinal muscular atrophy — have been approved for use in the U.S. A number of recent safety and manufacturing setbacks have also stymied progress, concerns highlighted at an FDA meeting in September.
The problem, the NIH said in a statement, is developing, testing and producing gene therapies is incredibly complex, time-consuming and expensive. The new consortium aims to tackle some of the most common issues facing gene therapy developers and create a "standardized therapeutic development model" that would help speed new treatments.
One of the main aims for the group will be better understanding the adeno-associated viral vectors, or AAVs, that are often used to deliver gene therapies into the body. The unknowns regarding the safety of AAVs were a focus at the FDA's gene therapy meeting, and the consortium plans to help through research into the basic biology of the vectors. The group will also develop a set of standard analytic tests for AAVs that, if put into practice, could make manufacturing more efficient.
The consortium will additionally look for ways to streamline the regulatory process by standardizing preclinical testing and shortening the path from animal research to studies in humans. And it will support four to six clinical trials of gene therapies in different rare diseases.
The effort comes as the FDA tries to come to grips with the growing number of setbacks gene therapy research has faced. Following its two-day meeting in September, the agency issued two guidance statements for the industry.
Funding for the new consortium is relatively modest compared with the billions of dollars being poured into gene therapy research and acquisitions in the industry. But each of the group’s partners brings its own expertise.
The NIH listed five nonprofit groups involved in the consortium: the Alliance for Regenerative Medicine, the American Society of Gene and Cell Therapy, CureDuchenne, the National Organization for Rare Disorders and the National Institute for Innovation in Manufacturing Biopharmaceuticals. On the industry side, Biogen, the Novartis Institutes for BioMedical Research, Takeda, Taysha Gene Therapies and Ultragenyx are also participating.