- Andelyn Biosciences chosen by UMass Chan Medical School to manufacture clinical grade AAV9-CSA vector for Cockayne syndrome treatment.
- Partnership with Riaan Research Initiative marks progress towards clinical trials for the fatal pediatric disorder.
Andelyn Biosciences, Inc., a Contract Development and Manufacturing Organisation (CDMO) specializing in cell and gene therapy, has been selected by UMass Chan Medical School to utilize its suspension AAV Curator™ Platform for manufacturing clinical grade AAV9-CSA vector aimed at treating Cockayne syndrome.
The decision follows a substantial donation from Riaan Research Initiative, the largest known contribution towards Cockayne syndrome treatment development. This partnership underscores the joint effort to accelerate the start of clinical trials, leveraging Andelyn’s expertise in gene therapy development and manufacturing to streamline production and drive program success.
Miguel Sena-Esteves, PhD, Associate Professor of Neurology and Director of the Translational Institute for Molecular Therapeutics at UMass Chan Medical School, expressed optimism, stating, “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.”
Matt Niloff, Chief Commercial Officer of Andelyn, emphasized the company’s commitment, saying, “We are honored to have been selected to be a key contributor in the advancement of this critical therapy for Cockayne syndrome.” He highlighted Andelyn’s scalable development and manufacturing capabilities across its Columbus, Ohio facilities, which are crucial for enabling life-altering cell and gene therapies.
Overview of outsourcing trends 
