other_material
confidence high
sentiment positive
materiality 0.85
PepGen reports 53.7% mean splicing correction in DM1 patients at 15 mg/kg dose
PepGen Inc.
- Mean splicing correction of 53.7% at 15 mg/kg (n=6) by 22-gene panel at Day 28; all 6 patients responded.
- PGN-EDODM1 well-tolerated; no serious treatment-related AEs, all mild/moderate, transient, resolved without intervention.
- Dose-proportional muscle concentration increase vs 5 mg/kg (12.3%) and 10 mg/kg (29.1%) cohorts.
- FREEDOM2 MAD study 5 mg/kg cohort data expected Q1 2026; 10 mg/kg cohort dosing to begin Q1 2026.
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