The Ohio State University has pioneered foundational, translational and clinical gene therapy platforms, and the university’s new Gene Therapy Institute will support the rapid expansion and utilization of gene therapy globally.
Gene therapy techniques are the result of decades of cross-disciplinary research that treat or prevent disease by replacing or correcting genetic alterations. Researchers, including leaders at Ohio State, are pioneering novel foundational and therapeutic approaches to address what were previously untreatable, or incompletely treated, life-threatening diseases.
Successful translation of gene therapy is a complex process spanning research, product development and production, quality control, clinical trials, regulatory assessment and public-private collaborations.
Ohio State’s Gene Therapy Institute will coordinate existing strengths to accelerate the expansion of gene therapies. Related research is being conducted by over 50 faculty across the colleges of Medicine, Arts and Sciences, Law, Business, Veterinary Medicine, Engineering and Pharmacy.
The university holds one of the largest first-in-human clinical trial portfolios: 10 active trials; grant support of $14.6 million for a first-in-human nervous system gene therapy trial and $4.8 million for translational CRISPR-gene therapy research from the National Institutes of Health; and research collaborations with industry partners that include Battelle, Medtronic, Biogen and Bayer.
“I am so pleased to announce that we are launching a new Gene Therapy Institute to benefit so many people around the globe suffering from diseases caused by missing or mutated genes,” said Ohio State President Kristina M. Johnson during her State of the University address on April 21. “Our new institute offers Ohio State the opportunity to be the world leader in gene therapy.”
The institute will be led by Dr. Russell Lonser, professor and chair of neurological surgery, and Dr. Krystof Bankiewicz, professor of neurological surgery, at the Ohio State College of Medicine, and will directly involve leaders, faculty, staff and trainees from across the university.
“The creation of this collaborative university institute will most effectively and swiftly advance gene therapy science – ultimately leading to advanced treatments for patients,” Lonser said.
The launch of the Gene Therapy Institute is part of the university’s strategic plan to increase convergent research opportunities and accelerate societal impact. The institute will report to the Enterprise for Research, Innovation and Knowledge.