PepGen gets UK MHRA clearance to start Phase 2 trial of PGN-EDO51 for DMD

PepGen Inc.

MHRA authorized CTA for CONNECT2-EDO51 Phase 2 trial of PGN-EDO51 in DMD patients amenable to exon 51 skipping.
Trial is randomized, placebo-controlled, multiple ascending dose, enrolling ~20 boys/young men aged ≥6 years.
PGN-EDO51 is an oligonucleotide therapy; data from CONNECT1 (ongoing Phase 1) plus CONNECT2 could support accelerated approval.
Starting dose 5 mg/kg escalating to 10 mg/kg and potentially higher; muscle biopsies at baseline and week 25 to assess exon skipping and dystrophin production.
Participants have option for open-label extension after 24-week treatment period.

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