PepGen reported new Phase 2 data for PGN-EDODM1 in myotonic dystrophy type 1 (DM1), showing dose-dependent splicing correction alongside improvements in mobility and grip strength. After 13 weeks of follow-up in the trial, treated patients averaged 46.5% splicing repair across six participants at the higher dosing regimen, with four patients exceeding 50%. The company said treatment-related adverse events were mild to moderate, with nausea as the most common event. PepGen also reported no evidence of cumulative kidney damage over the 16-week observation period, a key safety consideration for RNA-based therapies. For investors and competitors, the readout elevates PepGen’s differentiation versus earlier-generation approaches, while increasing scrutiny on durability, functional outcomes over longer follow-up, and translational safety monitoring.