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PepGen Inc. (NASDAQ:PEPG), a clinical-stage biotechnology company advancing the next generation of oligonucleotide therapies with the goal of transforming the treatment of severe neuromuscular and neurological diseases, today announced that the first patient has been dosed in its CONNECT1-EDO51 Phase 2, open-label multiple ascending dose (MAD) clinical trial evaluating PGN-EDO51 for the treatment of Duchenne muscular dystrophy (DMD) patients amendable to an exon 51 skipping therapy.
- Preliminary data from the 5 mg/kg PGN-EDO51 dose level in the CONNECT1-EDO51 Phase 2 trial are expected mid-2024: including initial safety, exon 51 skipping and dystrophin protein production data -
- Phase 1 results following a single dose of 10 mg/kg of PGN-EDO51 in healthy volunteers demonstrated peak exon 51 skipping of 3.8% with 100% of subjects demonstrating exon skipping -
Posted In: PEPG
