Nationwide Children’s Hospital Forms Gene Therapy Company, Andelyn Biosciences

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The formation of the new gene therapy company stems from the progress and success of Nationwide Children’s Hospital’s clinical manufacturing and gene therapy work.

Columbus, Ohio-based Nationwide Children’s Hospital announced on Jan. 13, 2020 that it has created Andelyn Biosciences, an affiliate company that will manufacture gene therapy products for the biotechnology and pharmaceutical industries. The new company is scheduled to begin operations in 2023 and will be central Ohio’s first commercial-scale good manufacturing practices (cGMP) clinical manufacturing facility devoted to gene therapies, according to Nationwide Children’s Hospital.

The formation of Andelyn Biosciences stems from the 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 recognized among the nation’s leaders in developing gene therapies, according to the hospital. For several years, AWRI has operated a small-scale facility on-site 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. Beginning in summer 2020, the current 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, chief scientific officer of AWRI, in a press release issued by Nationwide Children’s Hospital. “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. As a for-profit company, revenues generated by Andelyn Biosciences will allow us 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 finalized.


“Central Ohio is emerging as a life sciences hub and Andelyn Biosciences will create jobs and expand our region’s influence in the biotechnology sector, while accelerating breakthrough therapies,” said Severina Kraner, JobsOhio healthcare director, in the press release.

“Andelyn Biosciences is another powerful example of the ways Nationwide Children’s Hospital is advancing healthcare and innovation in the Columbus region. The impact they have had, and will continue to have, has proved invaluable to our economy, communities, and residents,” said Kenny McDonald, president and CEO of One Columbus, in the press release.

The name Andelyn Biosciences represents a hybrid of two pioneering gene therapy recipients who participated in pivotal Phase I clinical trials at AWRI, Andrew Kilbarger and Evelyn Villarreal. At eight years old, Kilbarger received the first US human investigational gene therapy for Duchenne muscular dystrophy in 2006. At only eight weeks old, Villarreal received investigational gene therapy for spinal muscular atrophy in 2015. “Andelyn” combines their names to represent all the families who have participated in the research that makes gene therapies possible today, Nationwide Children’s Hospital explained.

Source: Nationwide Children's Hospital