Nationwide Children’s Hospital Renames Center for Gene Therapy, Honoring Jerry Mendell, MD

(COLUMBUS, Ohio) - Nationwide Children's Hospital announced today the renaming and dedication of the Jerry R. Mendell, MD Center for Gene Therapy at the Abigail Wexner Research Institute, in honor of Jerry Mendell, MD, a pioneer in the fields of neurology and gene therapy and a catalyst for advancement in the treatment of multiple neuromuscular disorders.

Dr. Mendell's work in neuromuscular disease began more than 50 years ago, when he saw his first patient with Duchenne muscular dystrophy (DMD). Thanks in part to his passion to develop new treatments for patients with neuromuscular disorders, he led the pivotal clinical trials for two of the first eight gene therapies approved by the FDA. In 2019, Zolgensma® became the first systemically delivered gene therapy approved by the FDA, for children with spinal muscular atrophy (SMA) type 1. In 2023, ELEVIDYS® was granted an accelerated approval from the FDA, for children 4-5 years old with DMD, followed in June 2024 by a full approval for ambulatory and an accelerated approval for nonambulatory DMD patients. His team previously conducted the studies leading to approval of the first exon skipping therapy for DMD, an approach that has also evolved as a major treatment for DMD worldwide.

"It is an appropriate gesture to honor Dr. Mendell's decades of achievement in this manner, given the enormous impact he has had on the lives of children as well as physicians and scientists over the course of his career and for years to come," said Dennis Durbin, MD, MSCE, president of the Abigail Wexner Research Institute at Nationwide Children's. "Dr. Mendell led this center from its infancy with dedication and passion for the patients it serves. It is entirely fitting that the center now bears his name, signifying his foundational legacy where innovations from the center continue with the same fervor as when it was created."

Established in 2002, the Center for Gene Therapy aims to investigate and employ the use of gene -based therapeutics for prevention and treatment of human diseases. In addition to clinical trials in muscular dystrophies, the center has a range of programs directed toward central nervous system disorders. Center faculty are also evaluating the use of a variety of techniques, including delivery of surrogate genes, vectorized exon skipping, nonviral gene delivery, gene editing with CRISPR-based systems, modulation of gene expression, and fetal gene therapy approaches.

Dr. Mendell was elected to the National Academy of Medicine in 2021 and was the first recipient of an award named in his honor by the American Society for Gene and Cell Therapy, the Jerry Mendell Translational Medicine Award. He has published more than 400 articles and authored books on skeletal muscle disease, peripheral nerve disorders and gene therapy.

"I've devoted my life to developing therapies with the support of my wife and family that will improve the lives of children and their families. That work could only be accomplished with the support of a dedicated team of professionals and the pioneering spirit of the children and families who participate in clinical studies," said Dr. Mendell. "I am grateful to have been able to help establish a center for gene therapy at Nationwide Children's Hospital that will continue to change the lives of children well into the future."

Currently, Dr. Mendell serves as an advisor to the Abigail Wexner Research Institute at Nationwide Children's, as well as senior advisor at Sarepta Therapeutics. He is also an emeritus professor of pediatrics and neurology at The Ohio State University.

About The Abigail Wexner Research Institute at Nationwide Children's Hospital
Named to the Top 10 Honor Roll on U.S. News & World Report's 2023-24 list of "Best Children's Hospitals," Nationwide Children's Hospital is one of America's largest not-for-profit free-standing pediatric health care systems providing unique expertise in pediatric population health, behavioral health, genomics and health equity as the next frontiers in pediatric medicine, leading to best outcomes for the health of the whole child. Integrated clinical and research programs are part of what allows Nationwide Children's to advance its unique model of care. As home to the Department of Pediatrics of The Ohio State University College of Medicine, Nationwide Children's faculty train the next generation of pediatricians, scientists and pediatric specialists. The Abigail Wexner Research Institute at Nationwide Children's Hospital is one of the Top 10 National Institutes of Health-funded free-standing pediatric research facilities in the U.S., supporting basic, clinical, translational, behavioral and population health research. The AWRI is comprised of multidisciplinary Centers of Emphasis paired with advanced infrastructure supporting capabilities such as technology commercialization for discoveries; gene- and cell-based therapies; and genome sequencing and analysis. More information is available at NationwideChildrens.org/Research.

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