Andelyn Biosciences Selected by UMass Chan Medical School to Manufacture AAV9-CSA Vector for Cockayne Syndrome Treatment

11 July 2024 | Thursday | News

Partnership with Riaan Research Initiative and Andelyn Biosciences Brings Clinical Trials for Fatal Pediatric Disorder Closer to Reality, Leveraging Advanced AAV Curator™ Platform for Gene Therapy Development
Image Source : Public Domain

Image Source : Public Domain

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.



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