Ultragenyx joins FDA, NIH partnership to accelerate treatments for rare diseases
Ultragenyx recently joined other leaders from the public, private, and nonprofit sectors for a first-of-its-kind coalition to accelerate the development of gene therapies for patients with rare diseases. The Bespoke Gene Therapy Consortium (BGTC), launched by the National Institutes of Health (NIH) with the Food and Drug Administration (FDA), and managed by the Foundation for the National Institutes of Health (FNIH), is a collaboration dedicated to creating a standardized approach that will reduce up-front costs and lower barriers to developing new gene therapies for rare and ultra-rare diseases.
BGTC is a five-year, $76.5 million initiative emerging from the NIH Accelerating Medicines Partnership® (AMP®) Program. The consortium’s members include 11 NIH organizations, the FDA, five nonprofit and patient groups, and ten corporations. Ultragenyx joined the effort due to the strong alignment with the company’s mission and that of BGTC. BGTC aims to optimize and streamline the gene therapy development process to help fill the unmet medical needs of people with rare diseases. The consortium will work with researchers to develop improved insights into the biology, efficiency, and production of adeno-associated virus (AAV) vectors, the leading delivery platform for gene therapy.
“At Ultragenyx, we recognize both the life-changing potential that one-time gene therapies can have on people with rare diseases and the challenges associated with developing and manufacturing these innovative therapies – that’s why we are participating in the Accelerating Medicines Partnership Bespoke Gene Therapy Consortium,” said Reed Clark, Ph.D., senior vice president of research at Ultragenyx Gene Therapy. Clark added, “The goal of this partnership aligns with our mission to deliver safe and effective new treatments to patients with rare diseases. We look forward to collaborating with our peers and sharing our experience and insights in gene therapy to streamline the overall process and bring new bespoke gene therapies to patients with rare genetic diseases faster, safer and more efficiently.”
Consistent with Ultragenyx’s vision of the future of rare disease medicine, the company and its leadership team are committed to sharing their expertise to advance the industry and make more treatments available to patients around the world. For more information on the Bespoke Gene Therapy Consortium, please see the FNIH press release.
Jeff Blake is senior director of corporate communications at Ultragenyx