New Public-Private Consortium Seeks to Drive Gene Therapies for Rare Diseases

The Bespoke Gene Therapy Consortium, part of the National Institutes of Health’s Accelerating Medicines Partnership managed by the Foundation for the National Institutes of Health, unveiled itself with a plan to streamline the development of gene therapies to address rare diseases.

Photo: Joni Rutter, acting director of NIH’s National Center for Advancing Translational Sciences

It is the first Accelerating Medicines Partnership initiative focused on rare diseases.

You can listen to a recent RARECast with P.J. Brooks, deputy director of the Offices of Rare Disease Research at the National Center for Accelerating Translational Sciences (NCATS), where he discusses the creation of the Bespoke Gene Therapy Consortium.

The public-private partnership includes the National Institutes of Health, the U.S. Food and Drug Administration, 10 biopharmaceutical companies and five nonprofit organizations. NIH and private partners will contribute $76 million over five years to support BGTC-funded projects.

Participating NIH institutes and centers will provide a little more than half the funding, pending availability of funds. NCATS, which developed the related Platform Vector Gene Therapy (PaVe-GT) program, is the NIH lead institute for consortium.

“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 Rutter, acting director of NCATS. “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.”

While individual rare diseases affect small numbers of people, collectively they affect millions. Gene therapy represents a promising approach to treat and potentially cure disease caused by a mutation to a single gene, but development of these therapies is costly and complex. That’s in part due to limited access to tools and technologies, lack of standards across the field, and a one-disease-at-a-time approach to therapeutic development.

A primary aim of BGTC is to improve understanding of the basic biology of a common gene delivery vector known as the adeno-associated virus (AAV). BGTC researchers will examine the biological and mechanistic steps involved in AAV vector production, vector delivery of genes into human cells and how therapeutic genes are activated in target cells. These results will provide important information for improving the efficiency of vector manufacturing and enhancing the overall therapeutic benefit of AAV gene therapy. 

To improve and accelerate gene and vector manufacturing and production processes, the BGTC program will develop a standard set of analytic tests to apply to the manufacture of viral vectors made by consortium researchers. Such tests could be broadly applicable to different manufacturing methods and make the process of developing gene therapies for very rare conditions much more efficient.

A clinical component of BGTC-funded research will support between four and six clinical trials, each focused on a different rare disease. These diseases are expected to be rare, single-gene diseases with no gene therapies or commercial programs in development and that already have substantial groundwork in place to rapidly initiate preclinical and clinical studies. The trials will employ different types of AAV vectors that have been used before in clinical trials. For these trials, the BGTC will aim to shorten the path from studies in animal models of disease to human clinical trials.

The BGTC also will explore methods to streamline regulatory requirements and processes for the FDA approval of safe and effective gene therapies, including developing standardized approaches to preclinical testing.

“By leveraging on experience with a platform technology and by standardizing processes, gene therapy product development can be accelerated to allow more timely access to promising new therapies for patients who need them most,” said Peter Marks, director of FDA’s Center for Biologics Evaluation and Research. “FDA is committed to developing a regulatory paradigm that can advance gene therapies to meet the needs of patients with rare diseases.”

Private partners include Biogen, Janssen Research & Development, Novartis Pharma, Pfizer, RegenxBio, Spark Therapeutics, Takeda Pharmaceutical, Taysha Gene Therapies, Thermo Fisher Scientific, and Ultragenyx Pharmaceutical.

Non-profit partners include the Alliance for Regenerative Medicine, the American Society of Gene and Cell Therapy, CureDuchenne, National Institute for Innovation in Manufacturing Biopharmaceuticals, and the Organization for Rare Disorders.

In addition to NCATS, participating NIH institutes include the Eunice Kennedy Shriver National Institute of Child Health and Human Development; National Eye Institute; National Heart, Lung, and Blood Institute; National Human Genome Research Institute; National Institute of Arthritis and Musculoskeletal and Skin Diseases; National Institute of Dental and Craniofacial Research; National Institute of Mental Health; National Institute of Neurological Disorders and Stroke; and National Institute on Deafness and Other Communication Disorders. 

This article is part of our ongoing Platforms of Hope series, which is supported by Pfizer, Inc.,  Bluebird, and Novartis Gene Therapies. 

Author: Rare Daily Staff