Center for Gene Therapy, Nationwide Children's Hospital
The Nation's Premier Gene Therapy Hub Thrives at Nationwide Children's Hospital
Children with devastating neuromuscular disorders are finding hope through precise gene-based therapies. After decades of research into gene transfer, it is finally reaching the clinic in a meaningful way.
Over the past 30 years, the journey to gene therapy has been fraught with challenges. But in 2017, the field experienced exciting breakthroughs. No breakthrough received more attention than when researchers from Nationwide Children’s Hospital and The Ohio State University published the remarkable results of the phase 1 clinical trial of gene therapy for spinal muscular atrophy type 1 (SMA1) in the New England Journal of Medicine. On May 24, 2019, this therapy received approval from the FDA – only the second gene therapy approved in the US.
Beyond SMA, gene therapy solutions are being studied at Nationwide Children’s Center for Gene Therapy for myriad neuromuscular and neurological diseases, including Charcot-Marie-Tooth disease, Batten disease, and muscular dystrophies to name a few. The Center has a robust pipeline of more than 20 genetic targets for treating various disorders. A comprehensive infrastructure including a clinical manufacturing facility, regulatory support, and clinical research resources have made Nationwide Children’s the premier institution for gene-based therapies.