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STAT·24m ago·2 min read
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STAT+: PepGen says early trial for muscular dystrophy drug looks promising

PepGen says its candidate is correcting more of the genetic stutter found in myotonic dystrophy type 1.

Nov 10, 2026·read at STAT ↗

STAT PlusExclusive PepGen says early trial for muscular dystrophy drug looks promising Infusion for a common dystrophy form is one of several in development Manage alerts for this article Save this story for later Share this article By Jason MastNov. 10, 2026 General Assignment Reporter 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.

The biotech PepGen said Tuesday that its experimental drug had shown, by one key measure, the most promising early data yet in the race to treat a muscle disorder that affects over 100,000 people in the U.S. and Europe. PepGen is a latecomer to the fight to treat myotonic dystrophy type 1 (DM1), a condition that typically robs patients of muscle control and strength beginning around adolescence.

Tuesday’s data come from six patients, who were treated for 12 weeks. Advertisement Dyne Therapeutics is close to completing a pivotal trial for a DM1 drug. Novartis just announced that a DM1 drug it acquired in its $12 billion buyout of Avidity Biosciences failed to improve a key measure of muscle control in a Phase 3 trial, casting a pall over the field.

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Subscribe Log In Clinical trials, drug development, genetics, rare diseases Submit a correction requestReprints 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. Newsletter Your morning rundown of the science, politics, and money driving biotech today Recommended Pharma November 9, 2026 STAT Plus: FDA approves novel Bayer drug to reduce recurrence of stroke Biotech November 9, 2026 STAT Plus: FDA warns GLP-1 drugs carry potential risk of rare eye condition Advertisement The Readout November 9, 2026 STAT Plus: Gene therapy’s long-debated cancer risk materializes Matt's Take November 8, 2026 STAT Plus: Why the latest heart drug failures should make us all more skeptical of AI hype Politics November 7, 2026 STAT Plus: BIO joins the legal fight against Medicare pilot that ties drug prices to peer countries Subscriber Picks

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