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Biomedical

It’s in our DNA: Why the Columbus Region is a Hub for Gene Therapy Companies

Recent developments from gene therapy companies and the supporting ecosystem have set a new trajectory for life sciences in Central Ohio.

May 23, 2022 // By Matt McQuade

When Amgen selected the Columbus Region for its newest and most advanced biomanufacturing plant in 2021, you could forgive much of the life sciences industry outside of Ohio for being surprised at the choice. The state had not been an especially visible player among Big Pharma up until that point, at least to those based elsewhere. In particular, the exciting growth and attraction of gene therapy companies had flown under the radar despite several early successes in the field.

It’s amazing what a $365 million investment will do – the Amgen decision[SD1]  has invited a closer look at the thriving ecosystem already here, which includes industry giants like Cardinal Health, Abbott Nutrition, Sarepta Therapeutics and Battelle spin-out AmplifyBio; pharmaceutical manufacturers like Hikma and American Regent; and digital health startups like CoverMyMeds, Olive and Updox.

But it is the developments involving several gene therapy companies and local research institutions that have set Central Ohio on its highest trajectory yet for life sciences growth.


Woman in a clean room working on gene therapies at a facility in Columbus Region of Ohio.

World-Class Hospitals Catalyze Innovation

The Columbus Region’s rise as a center for gene therapy companies and expertise is driven by the advanced science inside its research hospitals. Foremost among them is Nationwide Children’s Hospital (NCH), a leader in gene therapy since 2002 when the hospital launched its Center for Gene Therapy. NCH’s Abigail Wexner Research Center developed Zolgensma as one of the few gene and cell-based therapeutics used to treat spinal muscular atrophy. The treatment was licensed to AveXis in 2013 and approved by the FDA in 2019.

Along with NCH, The Wexner Medical Center at The Ohio State University is one of only 25 national clinical sites plugged into the NeuroNEXT program — a network of institutions working to improve productivity and expedite therapy development for neurological disorders. In August 2020, the Columbus-based lab completed the first ever novel gene therapy brain infusion for a patient suffering from Parkinson’s disease. In the gene therapy space, proximity to world-class hospitals and educational institutions encourages more funding, talent and knowledge sharing. The Region’s life sciences industry can innovate rapidly and effectively thanks to the clustering effect the anchor healthcare system creates.

Nationwide Children’s Hospital in Columbus, Ohio is one of the early innovators in gene therapy science.

Gene Therapy Companies Spinoff & Accelerate A New Era of Growth

Cutting-edge research from local hospitals and partner institutions also supplies the foundation for startups and spinoffs in the Region. In 2020, the success of NCH’s R&D led to the launch of Andelyn Biosciences â€” a for-profit gene therapy company affiliate that will manufacture materials for global biotechnology and pharmaceutical clinical studies.

Before that, another of NCH’s spinoffs, Myonexus Therapeutics, was acquired by Sarepta Therapeutics in 2019, along with five experimental gene therapies. The following year, the global biotech company invested $30 million towards the opening of its Gene Therapy Center of Excellence â€” a new 85,000-square-foot facility right in the heart of the Columbus Region. Over the four decades since the gene therapy company’s founding, Sarepta has become a national leader in precision genetic medicine for rare neuromuscular diseases.

Forge Biologics closed a $40 million funding round in 2020 and another $80 million in 2022. The company’s 175,000 square foot Columbus Region facility, known as “The Hearth,” conducts end-to-end manufacturing of gene therapy programs for biotech and pharma clients. One of the programs they are developing is a novel AAV and umbilical cord transplant combination to treat Krabbe disease.

But a cluster of gene therapy companies is only as good as its talent, which is also quickly developing in the Columbus Region. Through a $100M pledge from JobsOhio in 2020, the state’s economic development agency, The Ohio State University and NCH are partnering on a new 270-acre, $1 billion+ Innovation Districtbringing together students, researchers, city leaders, Fortune 500 companies and new startups to spur growth in the STEM community.

For reference, The Ohio State University’s Biomedical Engineering program awarded 72 degrees to students in 2020, and in the last decade, over 8,700 degrees in biology, biomedical science, chemistry and pharmacy have been granted throughout the Region. The Innovation District intends to triple STEM degrees over the next 15 years.

Life sciences and gene therapy companies looking to influence the field can benefit from the Region’s cutting-edge experience, dedicated labor market and strong private/public partnerships. Contact us today to learn how we can guide your company through the location decision process.


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Article // Biomedical


Matt McQuade serves as the Managing Director, Business Development for One Columbus.