PepGen announces positive Phase 2 data for PGN-EDO51 in DMD: exon skipping 2.15%, dystrophin increase

PepGen Inc.

PGN-EDO51 5 mg/kg achieved mean exon skipping of 2.15% in biceps tissue at week 13 (n=3).
Mean muscle-adjusted dystrophin level of 1.49% of normal, +0.70% from baseline; absolute dystrophin 0.61%, +0.26%.
Drug well tolerated; no discontinuations, one mild AE resolved, no kidney or electrolyte issues.
All three patients continue in long-term extension; 10 mg/kg cohort dosing ongoing with results expected in early 2025.
CONNECT2 Phase 2 trial open in UK; US site expected by year-end; design being optimized based on data.

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