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

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.

“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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