STAT+: Regenxbio says Duchenne gene therapy succeeded in clinical trial, paving way for FDA submission

STAT PlusBiotech Regenxbio says Duchenne gene therapy succeeded in clinical trial, paving way for FDA submission Company hopes to prove treatment is more effective, safer than Sarepta Therapeutics’ Elevidys Manage alerts for this article Email this article Share this article By Adam Feuerstein and Jason MastMay 14, 2026 Adam Feuerstein[email protected]Adam Feuerstein is a senior writer and biotech columnist, reporting on the crossroads of drug development, business, Wall Street, and biotechnology. He is also a co-host of the weekly biotech podcast The Readout Loud and author of the newsletter Adam’s Biotech Scorecard. You can reach Adam on Signal at stataf.54.Jason Mast[email protected]Jason is a general assignment reporter, with particular focus on genetic medicine and rare disease.

Confidential tips can be sent on Signal at JasonMast.77. Regenxbio said Thursday that its experimental gene therapy for Duchenne muscular dystrophy produced sufficiently high levels of a miniaturized muscle protein broken in the fatal neuromuscular disease, paving the way for a submission to the Food and Drug Administration. The company is seeking to create a Duchenne gene therapy that is more effective and safer than Sarepta Therapeutics’ Elevidys, which has been hampered by safety concerns, particularly following the deaths of two recipients from liver failure.Advertisement “I think our data checks every single box that you would want for accelerated approval,” Regenxbio CEO Curran Simpson told STAT.

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Subscribe Log In biotechnology, drug development, Pharmaceuticals, rare diseases, STAT+ Submit a correction requestReprints Adam Feuerstein Senior Writer, Biotech Adam Feuerstein is a senior writer and biotech columnist, reporting on the crossroads of drug development, business, Wall Street, and biotechnology. He is also a co-host of the weekly biotech podcast The Readout Loud and author of the newsletter Adam’s Biotech Scorecard. You can reach Adam on Signal at stataf.54.

Jason Mast General Assignment Reporter Jason is a general assignment reporter, with particular focus on genetic medicine and rare disease. Confidential tips can be sent on Signal at JasonMast.77. STAT+ Newsletter Senior writer Adam Feuerstein's unfiltered, uncompromising analysis on the biotech world Recommended Exclusive May 14, 2026 STAT Plus: CREATE Medicines, a biotech company developing CAR-T therapies, raises $122 million Adam's Biotech Scorecard May 14, 2026 STAT Plus: ‘No drama’ Diamantas should serve biotech well as acting FDA chief Advertisement Exclusive May 13, 2026 STAT Plus: What the Trump administration wants in its next FDA leader The Readout May 13, 2026 STAT Plus: Gene therapy viruses linked to a boy’s tumor Biotech May 13, 2026 STAT Plus: Drug meant to make gene therapy safer may also make it less effective Subscriber Picks