Biotech pipeline – RNA-based therapy for myotonic dystrophy shows splicing correction in Phase 2

PepGen said its Phase 2 trial of PGN-EDODM1 in myotonic dystrophy type 1 (DM1) showed substantial correction of splicing errors after 13 weeks of follow-up. The company reported an average 46.5% reduction in mis-splicing errors across six treated patients, with four patients exceeding 50%, alongside improvements in mobility- and muscle-strength–related assessments. PepGen characterized treatment-related adverse events as mild to moderate, with nausea as the most common event. The company also reported no evidence of cumulative kidney damage over the 16-week observation period, a key concern for systemic RNA-based therapies.