The Research Institute at Nationwide Children’s Hospital Spins Off Groundbreaking Gene Therapy Companies

The resources and expertise of Ohio’s renowned research and healthcare institutions drive discoveries that could improve millions of lives.

Nationwide Children’s Hospital in Columbus, Ohio, is one of the leading pediatric hospitals in the nation. In 2017, the hospital had over 1.4 million patient visits from all 50 states as well as 48 countries. Its nonprofit research entity, the Research Institute, is a top-funded pediatric research institution with more than 1,300 employees and investigators. The Research Institute at Nationwide Children’s Hospital occupies over 500,000 square feet of dedicated space and has 15 multidisciplinary Centers of Emphasis, one of which is the Center for Gene Therapy.

The Center for Gene Therapy has grown rapidly since the early 2000s. Nationwide’s leadership, including Dr. John Barnard, president of the Research Institute, Dr. Jerry Mendell, principal investigator in the Center for Gene Therapy, and Dr. Brian Kaspar, a former member of the center, have advocated for the continuation of research over the years. Today, these individuals are some of the most influential minds in gene therapy.

In addition to basic science expertise, the Research Institute has the internal infrastructure and resources to support translation. In particular, a Good Manufacturing Practice (GMP) facility, built in 2010, has in-house regulatory services to engage the Food and Drug Administration as well as a fully integrated clinical research services team which make it possible to take discoveries from bench to bedside.

A tipping point for the Center for Gene Therapy came in 2013 when AveXis, one of its promising startups, began clinical trials. Data from the therapy’s success led to the company’s acquisition by Novartis in 2018 for $8.7 billion. Since then, the Research Institute has spun out four additional gene therapy companies and won the attention of leading biotechnology companies, solidifying its role as a leader in gene therapy.

The dedication to improving the lives of children and their families fueled Nationwide Children’s Hospital toward becoming a global healthcare leader.

The continued success from the Center for Gene Therapy has produced five startups, including AveXis, along with other partnerships and licensing deals:

• Myonexus Therapeutics Inc.: Focuses on rare genetic diseases, including the first treatments for limb-girdle muscular dystrophy (LGMD). Sarepta Therapeutics Inc. partnered with Myonexus Therapeutics by investing up to $105 million and has an exclusive option to acquire the company.
• Abeona Therapeutics: Develops gene therapy for potential treatment of individuals with rare diseases, including lysosomal storage diseases
• Celenex Inc.: Pursues treatment for rare, fatal neurological conditions. Amicus Therapeutics Inc. acquired the company last September with an upfront payment of $100 million.
• Milo Biotechnology: Advances therapies that increase muscle strength and improve the lives of patients with muscular dystrophy.