PepGen announced that the U.S. Food and Drug Administration granted both orphan drug and rare pediatric disease designations for PGN-EDO51, an investigational therapeutic for Duchenne muscular dystrophy patients whose mutations are amenable to an exon 51 skipping approach. PepGen is evaluating PGN-EDO51 for the treatment of DMD in the ongoing CONNECT1 Phase 2 trial, and expects to begin enrolling patients in the CONNECT2 Phase 2 trial later this year.
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