Andelyn Biosciences, a 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 experience in gene therapy development and manufacturing will be leveraged to industrialize the production process and drive program success.

“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,” said Miguel Sena-Esteves, Associate Professor of Neurology and Director of the Translational Institute for Molecular Therapeutics. “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, said, “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 scalable end-to-end development and manufacturing capabilities across its three Columbus, OH facilities, Andelyn continues to partner with innovator organizations to enable the progression of life-altering cell and gene therapies to cure rare and prevalent diseases.