PepGen Inc. Reports Unprecedented Splicing Correction in DM1 Patients with PGN-EDODM1 in Phase 1 Trial
Reuters
Sep 25
PepGen Inc. Reports Unprecedented Splicing Correction in DM1 Patients with PGN-EDODM1 in Phase 1 Trial
PepGen Inc. announced positive clinical data from the 15 mg/kg dose cohort of its ongoing FREEDOM-DM1 Phase 1 single ascending dose (SAD) study evaluating PGN-EDODM1 in patients with myotonic dystrophy type 1 (DM1). The results showed a mean splicing correction of 53.7% following a single dose, with all patients in the cohort demonstrating improved splicing. PGN-EDODM1 was generally well-tolerated, with drug-related adverse events reported as mild or moderate. The company stated that these data build upon previously reported results from lower dose cohorts and that data from the first cohort of the FREEDOM2 multiple ascending dose (MAD) study are expected in the first quarter of 2026. A copy of the corporate presentation including these results will be made available on the PepGen investor website.
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