How Nationwide Children's quietly became a leader in gene therapy

Nationwide Children's Hospital in Columbus, Ohio, has quietly become a go-to hub for gene therapy research and production, according to Xconomy National.

Since the start of 2018, the hospital has signed 28 biotechnology licensing deals, which is a large amount for a hospital that had barely in-house research two decades ago, according to the report.

Earlier in 2019, a Massachusetts-based biotech Sarepta bought a startup that spun out of Nationwide. The deal gave Sarepta access to five experimental gene therapies developed by researchers at the hospital. Prior to this deal, the Massachusetts biotech had already struck a deal to buy three other potential gene therapies from the hospital.

After flying under the radar for years, the number of potential gene therapies being produced at Nationwide has put the hospital "squarely on the national map," according to the report.

Nationwide's research hub for gene therapies took off when other researchers in the field were shying away after an infamous death during a gene therapy clinical trial in 1999. Jesse Gelsinger, an 18-year-old with a genetic liver disease, was the first person publicly identified as having died in a clinical trial for gene therapy after he suffered a massive immune response that led to organ failure and brain death.

"It looked like [gene therapy] had no future, people were really frightened," Jerry Mendell, MD, the director of Nationwide's gene therapy center and a pioneer in the field of neuromuscular disease research, told Xconomy.

Despite the scare, Nationwide felt that gene therapy would play an important role in modernizing medicine. They forged on with gene therapy research.

Now, one of the treatments developed by Dr. Mendell's team could soon become one of the few gene therapies approved for use in the U.S. Novartis purchased rights for Zogensma, a spinal muscular atrophy drug for $8.7 billion and expects an FDA decision on the drug in May.

"What we started here has had an incredible impact," Dr. Mendell told Xconomy.

Read the full report here.


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