Andelyn Biosciences, a CDMO involved in patient-focused cell and gene therapy, has been selected to manufacture adeno-associated vector (AAV) therapies in its suspension platform for several programs under the Foundation for the National Institutes of Health (FNIH) Accelerating Medicines Partnership® (AMP®) Bespoke Gene Therapy Consortium (BGTC).

BGTC has chosen to focus on eight rare disease programs with Andelyn selected to optimize and scale the AAV therapy processes for the treatments of CNGB1 Retinitis Pigmentosa 45 and NPHP5 retinal degeneration. Managed by the FNIH, AMP BGTC is a public-private partnership between the NIH, the FDA, biopharmaceutical and life science companies, and nonprofit and other organizations with a mission to speed the development and delivery of AAV gene therapies that could treat the millions of people affected by rare diseases.

“As a long-standing pioneer in AAV gene therapies with a focus on patients, we have great synergies with the BGTC,” commented Matt Niloff, Andelyn’s CCO. “Andelyn is privileged to work with the FNIH/BGTC on its first AMP which was established specifically to help overcome the major obstacles related to developing gene therapies for rare diseases.”

“This is an important step toward reaching our ultimate goal of getting gene therapies to rare disease patients in need sooner, and with fewer hurdles along the way,” added Courtney Silverthorn, associate VP for science partnerships at the FNIH and director of the AMP program.”

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