PepGen doses first patient in Phase 2 CONNECT1-EDO51 trial for Duchenne muscular dystrophy

PepGen Inc.

First patient dosed in open-label, multiple ascending dose Phase 2 trial of PGN-EDO51 in DMD patients amenable to exon 51 skipping.
Trial enrolls ~10 male patients aged >=8 years; starting dose 5 mg/kg escalating to 10 mg/kg pending safety.
Preliminary data from the 5 mg/kg cohort expected mid-2024: safety, exon skipping, and dystrophin production.
Phase 1 healthy volunteers showed peak exon 51 skipping of 3.8% after single 10 mg/kg dose; 100% had skipping.
PGN-EDO51 was generally well-tolerated through 15 mg/kg in Phase 1; trial ID NCT06079736.

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