PepGen has announced that the first participant has been dosed in its Phase 2 CONNECT1-EDO51 study, which will evaluate the safety and tolerability of PGN-EDO51, the company’s next-generation conjugated-PMO exon skipping therapy for the treatment of Duchenne patients amenable to exon 51 skipping.

The trial is expected to enroll 10 participants who are at least 8 years of age and amenable to an exon 51 skipping approach. In addition to safety, oligonucleotide muscle concentrations, exon skipping and dystrophin protein production in muscle will be assessed at week 12 following 4 monthly doses of PGN-EDO51.

PepGen anticipates reporting out the safety, exon skipping and dystrophin production from Cohort 1 mid-2024. The company indicated that the starting dose will escalate from 5 mg/kg to 10 mg/kg for Cohort 2, with further dose escalation to be determined based on evaluation of safety data from prior dose cohorts.

We look forward to continued updates from PepGen as the trial progresses.

Read PepGen’s press release here.