Nationwide Children’s Hospital has made competition for pharmaceutical companies by forming an affiliate company that will commercially manufacture gene therapy products. The new Andelyn Biosciences, scheduled to begin operations in 2023, will be central Ohio’s first commercial-scale cGMP clinical manufacturing facility devoted to gene therapies.
Andelyn Biosciences represents an important evolution of Nationwide Children’s Hospital’s current success in clinical manufacturing and gene therapy. The Abigail Wexner Research Institute (AWRI) at Nationwide Children’s is now widely recognised among the nation’s leaders in developing gene therapies, which are becoming safe and effective treatments for previously fatal or untreatable genetic diseases.
Getting to this point
For several years, AWRI has operated a small-scale onsite facility focused on the manufacture of early-stage gene therapy products for Phase I and II clinical trials. The current facility operates according to FDA cGMP guidelines for early stage clinical trials to ensure the safety of manufactured biologic products.
Andelyn Biosciences will be established as a for-profit subsidiary company and will continue current GMP operations. Then beginning summer 2020, this facility will manufacture products compliant with phase III clinical trial regulations while the new commercial manufacturing site is built and validated.
“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, MD, MSCE, 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.”
Durbin explained that as a for-profit company, revenues generated by Andelyn Biosciences will allow the organisation to reinvest back into the non-profit research mission at AWRI, supporting its commitment to advance best outcomes for children around the world.
To make Andelyn Biosciences possible, Nationwide Children’s is working with key partners including JobsOhio, the City of Columbus, One Columbus and Rev1 Ventures to create jobs and expand central Ohio’s influence in the biotechnology sector.
The Ohio State University’s West Campus innovation district is being explored as a location for Andelyn Biosciences, although an exact location has yet to be finalised.
Nomenclature
The name Andelyn Biosciences speaks to the company’s prime mission, representing a hybrid of two pioneering 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 8 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.
