UMass Chan Chooses Andelyn Biosciences to Produce AAV9-CSA Vector for Cockayne Syndrome

COLUMBUS, Ohio, July 10, 2024-- Andelyn Biosciences, Inc., a prominent cell and gene therapy Contract Development and Manufacturing Organization (CDMO), has been chosen by UMass Chan Medical School to utilize its suspension AAV Curator™ Platform for the production of clinical grade AAV9-CSA vector aimed at treating Cockayne syndrome.

The Riaan Research Initiative, a nonprofit patient-advocacy organization, has made the largest known donation to support the development of a treatment for Cockayne syndrome. This significant contribution, along with the collaboration with Andelyn, brings the initiation of clinical trials for this severe autosomal recessive disorder closer to reality. Andelyn’s extensive expertise in gene therapy development and manufacturing will be crucial in industrializing the production process and ensuring the program's success.

Miguel Sena-Esteves, PhD, Associate Professor of Neurology and Director of the Translational Institute for Molecular Therapeutics, expressed that the goal of making a difference for patients with Cockayne syndrome is progressively becoming achievable. "Signing the contract with Andelyn Biosciences to produce the clinical material marks a significant milestone towards our objective. Developing a gene therapy for fatal pediatric neurological diseases presents numerous challenges, but with the support of Riaan Research Initiative, we have assembled an exceptional team to advance our program to a first-in-human gene therapy clinical trial for Cockayne syndrome."

Matt Niloff, Chief Commercial Officer of Andelyn, stated, "We are honored to play a key role in advancing this critical therapy for Cockayne syndrome. With many organizations across the industry adopting our data-driven and configurable AAV Curator™ Platform, we are thrilled to welcome UMass as a vital partner utilizing the platform to expedite the Cockayne syndrome program."

Andelyn Biosciences boasts exceptional quality and scalable end-to-end development and manufacturing capabilities across its three facilities in Columbus, Ohio. The company continues to collaborate with leading innovator organizations to progress life-altering cell and gene therapies aimed at curing both rare and common diseases.

Andelyn Biosciences, Inc. is a comprehensive cell and gene therapy CDMO specializing in the development, characterization, and production of viral vectors for gene therapy. With over 20 years of experience, Andelyn's profound scientific expertise has facilitated the production of cGMP material for more than 450 clinical batches and 75 global clinical trials. The company operates out of three facilities in Columbus, Ohio, supporting clients in developing cell and gene therapies from initial concept through plasmid engineering and manufacturing, process and analytical development, and cGMP clinical and commercial manufacturing. Andelyn accelerates programs and delivers high-quality products through its configurable, data-driven AAV Curator™ Platform, or via tech transfers of established client programs. Their capabilities include cGMP manufacturing for both adherent and suspension processes up to a 2,000-liter capacity. A stringent quality system, regulatory support, and supply chain vertical integration further aid clients in bringing their critical therapies to market.

UMass Chan Medical School is one of the five campuses of the University of Massachusetts system. It includes the T.H. Chan School of Medicine, the Morningside Graduate School of Biomedical Sciences, the Tan Chingfen Graduate School of Nursing, ForHealth Consulting, a public service health care consulting division, and MassBiologics, the only nonprofit, FDA-licensed manufacturer of vaccines, biologics, and viral vector gene therapies in the United States. UMass Chan is committed to advancing together to improve health and wellness in diverse communities throughout Massachusetts and globally by leading and innovating in education, research, health care delivery, and public service. UMass Chan is recognized as a world-class destination for biomedical research, with over $300 million in annual funding and more than 500 active clinical trials. It is also a leader in primary care education, consistently ranked in the top 10 percent of medical schools for primary care by U.S. News and World Report. In 2021, the Medical School received a $175 million donation from The Morningside Foundation and was renamed UMass Chan Medical School.

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