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Endpoints·May 14·1 min read
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Regenxbio hits Duchenne gene therapy milestone, eyes 2027 FDA approval

Regenxbio said Thursday that its Duchenne muscular dystrophy gene therapy met the bar in a pivotal study, and it’s aiming for FDA approval in 2027. The biotech hopes to be the second company to commercialize ...

May 14, 2026·read at Endpoints ↗

Regenxbio CEO Curran Simpson at Endpoints' #JPM26 event (Brian Benton Photography) May 14, 2026 07:00 AM EDT Cell/Gene TxFDA+ Re­genxbio's Duchenne study suc­ceed­ed. Will it be enough to con­vince the FDA? Lei Lei Wu News Reporter Re­genxbio said Thurs­day that its Duchenne mus­cu­lar dy­s­tro­phy gene ther­a­py met the bar in a piv­otal study, and it’s aim­ing for FDA ap­proval in 2027.

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Reporting by Endpoints.

read at Endpoints ↗
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Approval4Duchenne Muscular Dystrophy4Gene Therapy (AAV)2

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