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PepGen Reports 46.5% Mean Splicing Correction in Phase 2 DM1 Trial

PUBT·11/10/2026 12:06:26
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PepGen Reports 46.5% Mean Splicing Correction in Phase 2 DM1 Trial
  • PepGen announced positive Phase 2 FREEDOM2-DM1 results for PGN-EDODM1 in myotonic dystrophy type 1 patients receiving 10 mg/kg.
  • Treatment improved disease-related splicing abnormalities and muscle function; patient-reported outcomes were inconclusive.
  • Reported adverse events were mild or moderate, including mild kidney-related events, with no serious adverse events or treatment discontinuations.
  • The 12.5 mg/kg cohort is fully enrolled, with results expected in the first half of 2027.
  • Open-label extension results are expected in January 2027, covering clinical data at 5 mg/kg and safety data at 10 mg/kg.


Disclaimer: This news brief was created by Public Technologies (PUBT) using generative artificial intelligence. While PUBT strives to provide accurate and timely information, this AI-generated content is for informational purposes only and should not be interpreted as financial, investment, or legal advice. PepGen Inc. published the original content used to generate this news brief via Business Wire (Ref. ID: 20261110113813) on November 10, 2026, and is solely responsible for the information contained therein.