Gene therapy invented in Columbus under scrutiny after patient deaths

The FDA gave a black box safety warning for a therapy that was invented right here in Columbus.

Sarepta Therapeutics’ gene therapy for treating the rare and fatal Duchenne Muscular Dystrophy was invented at the Abigail Wexner Research Institute at Nationwide Children’s Hospital in Columbus, OH. It’s called Elevidys and received FDA approval in 2023.

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But now, the FDA announced a serious warning that it could contribute to fatal liver injury and failure, and three patients have died after receiving it this year alone.

Sarepta, which opened a lab near Easton in 2021, was pressured by the FDA to stop shipping Elevidys, and they initially refused but then pulled it from the market. However, the FDA then said Sarepta could resume shipping. As of now, Elevidys still has the black box warning.

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