Rare Daily Staff
Nationwide Children’s Hospital said it has launching an affiliate company dubbed Andelyn Biosciences that will manufacture gene therapy products for the biotechnology and pharmaceutical industry.
Andelyn is expected to begin operations in 2023. It will be the first commercial-scale Good Manufacturing Practices clinical manufacturing facility devoted to gene therapies in central Ohio.
Nationwide said Andelyn Biosciences represents an “important evolution” of its success in clinical manufacturing and gene therapy. The Abigail Wexner Research Institute at Nationwide Children’s has operated a small-scale facility on-site focused on the manufacture of early-stage gene therapy products for phase 1 and 2 clinical trials. The current facility operates according to Food and Drug Administration cGMP guidelines for early stage clinical trials to ensure the safety of manufactured biologic products.
“We are on the cutting edge of pediatric gene therapy, and clients from around the world currently access AWRI’s knowledge, resources, experience and technology,” said Dennis Durbin, chief scientific officer of AWRI. “The goal of Andelyn Biosciences is to support the advancement of novel gene therapies for rare genetic diseases by building commercial manufacturing capacity which is needed as more of these treatments are developed over the coming years.”
Andelyn Biosciences will be established as a for-profit subsidiary company and will continue current GMP operations; beginning this summer, this facility will manufacture products compliant with phase 3 clinical trial regulations while the new commercial manufacturing site is built and validated.
The name Andelyn Biosciences is derived from two gene therapy recipients who participated in pivotal phase I clinical trials at AWRI. Andrew Kilbarger was 8 years old when he received the first U.S. human investigational gene therapy for Duchenne muscular dystrophy in 2006. Evelyn Villarreal was only eight weeks old when she received investigational gene therapy for spinal muscular atrophy in 2015. “Andelyn” combines their names to represent all the families who have courageously participated in the research that makes today’s gene therapies possible.
Photo: Dennis Durbin, chief scientific officer of Abigail Wexner Research Institute