Gene Therapy

The Cures of Tomorrow Start in Ohio

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Cutting-edge research, investment and commercialization in Ohio drive a unique gene therapy ecosystem

Anchored by the Research Institute at Nationwide Children’s Hospital and The Ohio State University, both in Columbus, the state of Ohio has become fertile ground for the rapidly evolving gene therapy industry. World-class researchers, high-tech facilities and a growing investor base fuel an ecosystem that is creating startups and attracting both entrepreneurs and international investment to highly regarded healthcare hubs built around the Cleveland Clinic in Northeast Ohio, Cincinnati Children's hospital in Southwest Ohio, and Columbus. As a result, Ohio’s rapidly evolving gene therapy sector is attracting global attention as it commercializes the cures of the future.

“There’s a rare combination of resources in Ohio that make it a really attractive destination for gene therapy companies. Entrepreneurs and young biotechs can tap into clinical expertise at The Research Institute at Nationwide Children’s Hospital, and their resources like the GMP facility and in-house regulatory group. In addition, forming a company in Ohio, where there is more risk tolerance and great early stage funding partners, gives you a chance to excel as an entrepreneur much quicker.”
—Al Hawkins, founder and CEO, Milo Biotechnology

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Consider the following aspects of Ohio’s growing gene therapy ecosystem:

  • Nearly $10 billion of investment in gene therapy startups spinning out of Nationwide Children’s Hospital since 2018
  • Ohio is tied for 4th among states in number of identified gene therapy startups and companies.
  • Its hospitals have some of the most advanced research and development facilities in the nation and attract top researchers from around the world.
  • The ecosystem attracts established companies from across the country. Sarepta and Abeona Therapeutics are investing in Ohio; Novartis and Amicus acquired companies created in Ohio.
  • The Research Institute at Nationwide Children’s Hospital is one of the top 10 National Institutes of Health (NIH) funded freestanding pediatric research facilities in the U.S.
  • Academic research partnerships with large organizations such as Nationwide Children’s and The Ohio State University or Cincinnati Children’s and the University of Cincinnati. These relationships build the talent pipeline in Ohio to support the state’s growing gene therapy sector.
  • A maturing research infrastructure in gene therapy developed over two decades that includes the nationally-renowned Research Institute at Nationwide Children’s Hospital. The Institute has vertically integrated early stage gene therapy R&D capability.
  • A relatively low cost of living and high quality of life.

Ohio’s gene therapy ecosystem is generating momentum in a swiftly growing sector. Wherever your dream is on the journey to commercialization, Ohio’s gene therapy assets can accelerate your growth.

MIT Technology Review

Two sick children and a $1.5 million bill: One family’s race for a gene therapy cure

Nationally recognized Nationwide Children's Hospital in Columbus, Ohio is home to some of the most promising gene-therapy trials and research. Read more about their gene therapy work in this October 2018 article from the MIT Technology Review.

Testimonials from Gene Therapy Leaders

“We’re making a very significant commitment and investment in Columbus. We like to believe with the work that Nationwide Children’s Hospital has already done, coupled with our commitment, there is a real chance Columbus, Ohio, could become the most important place in the world for gene therapy development.”
—Doug Ingram, president and CEO, Sarepta


“Nationwide Children’s Hospital has strategically developed a fully integrated, early stage gene therapy R&D capability, and, as a result, is truly differentiated in the emerging gene therapy sector. NCH has world class science, state of the art facilities, regulatory know-how, manufacturing expertise and capacity, and a pipeline of talent in collaboration with Ohio State University. Essentially, an early stage gene therapy biotech within a leading academic research hospital, NCH is making a positive impact in the lives of patients worldwide suffering from devastating genetic diseases.”
—Michael Triplett, PhD , chief executive officer, Myonexus



Tyler Allchin
Tyler Allchin