Andelyn Biosciences Selected by UMass Chan Medical School to Manufacture Clinical Grade AAV9-CSA Vector to Treat Cockayne Syndrome

Media ReleaseNewsJuly 10, 2024

COLUMBUS, OH, July 10, 2024 Andelyn Biosciences, Inc., a leading and patient-focused cell and gene therapy Contract Development and Manufacturing Organization (CDMO), has been selected by UMass Chan Medical School to use its suspension AAV Curator™ Platform to manufacture clinical grade AAV9-CSA vector to treat Cockayne syndrome.

The nonprofit patient-advocacy organization, Riaan Research Initiative, has provided the largest known donation ever made toward the development of a treatment for Cockayne syndrome.  The gift from Riaan Research Initiative and the partnership with Andelyn brings the start of clinical trials for the fatal autosomal recessive disorder one step closer.  Andelyn’s extensive experience in gene therapy development and manufacturing will be leveraged to industrialize the production process and drive program success.

Miguel Sena-Esteves, PhD, Associate Professor of Neurology and Director of the Translational Institute for Molecular Therapeutics said, “Our dream of making a difference for Cockayne patients is coming closer every day, and signing the contract with Andelyn Biosciences to make the clinical material is a major step in that direction. Developing a gene therapy for fatal pediatric neurological diseases is challenging in many ways, but together with Riaan Research Initiative we have an outstanding team to bring our program to a first-in-human gene therapy clinical trial for Cockayne syndrome”.

Matt Niloff, Chief Commercial Officer of Andelyn, adds, “We are honored to have been selected to be a key contributor in the advancement of this critical therapy for Cockayne syndrome.  With more and more organizations across the industry adopting our data-driven and configurable AAV Curator™ Platform, we are excited to add UMass as an important partner leveraging the platform to drive the acceleration of the Cockayne syndrome program.”

With exceptional quality and scalable end-to-end development and manufacturing capabilities across its three Columbus, Ohio facilities, Andelyn continues to partner with premier innovator organizations to enable the progression of life-altering cell and gene therapies to cure rare and prevalent diseases.


About Andelyn Biosciences, Inc.
Andelyn Biosciences is a full-service cell and gene therapy CDMO focused on the development, characterization and production of viral vectors for gene therapy. With more than 20 years of experience, Andelyn’s deep scientific expertise has resulted in the production of cGMP material for more than 450 clinical batches and 75 global clinical trials. Operating out of three Columbus, Ohio facilities, Andelyn supports its clients in developing cell and gene therapies from concept through plasmid engineering and manufacturing, process and analytical development, and cGMP clinical and commercial manufacturing. Andelyn accelerates programs and delivers high quality products by manufacturing product on its configurable, data-driven AAV Curator™ Platform, or through a tech transfer in of established client programs. Capabilities include cGMP manufacturing for both adherent and suspension processes up to a 2,000-liter capacity. A rigorous quality system, regulatory support and supply chain vertical integration further support clients in bringing their critical therapies to market. For more information, visit

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About UMass Chan Medical School
UMass Chan Medical School, one of five campuses of the University of Massachusetts system, comprises the T.H. Chan School of Medicine; the Morningside Graduate School of Biomedical Sciences; the Tan Chingfen Graduate School of Nursing; ForHealth Consulting of UMass Chan Medical School, 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. At UMass Chan, we are advancing together to improve the health and wellness of our diverse communities throughout Massachusetts and across the world by leading and innovating in education, research, health care delivery and public service. UMass Chan has built a reputation as a world-class destination for biomedical research, with more than $300 million in annual funding and more than 500 active clinical trials and as a leader in primary care education, perennially 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. Learn more at

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