PepGen doses first patient in Phase 1 FREEDOM-DM1 trial

PepGen announced that the first patient has been dosed in its Phase 1 clinical trial, FREEDOM-DM1, evaluating PGN-EDODM1 for the treatment of DM1. The FREEDOM-DM1 clinical trial is a randomized, placebo-controlled, single ascending dose study, intended to enroll approximately 24 adult patients with DM1 to evaluate the safety and tolerability of PGN-EDODM1. In addition to safety, oligonucleotide muscle concentrations and PGN-EDODM1 correction of mis-splicing of transcripts, clinical outcomes impacted in DM1 will also be assessed at 28 days and at later time points following a single dose of PGN-EDODM1. Per the protocol, the dose will escalate from the starting dose of 5 mg/kg to 10 mg/kg and then 20 mg/kg. Each dose escalation will be determined based upon evaluation of safety data from the prior dose cohorts.