Defeat Duchenne Canada is pleased to share news from PepGen, announcing the first participant dosed in a Phase 1 Clinical Trial of PGN-EDO51, an exon-skipping treatment of Duchenne muscular dystrophy, targeting Exon 51. This Phase 1 Healthy Normal Volunteer (HNV) study will evaluate safety and tolerability as the primary objective and will begin to evaluate the delivery and target engagement of PGN-EDO51 to enable subsequent patient trials.
“DMD is a devastating rare disease that places a significant burden on the lives of patients and their families. There are limited therapeutic options available, and our mission is to deliver a life-changing therapeutic to this community in need,” stated James McArthur, Ph.D., President and CEO of PepGen. “The initiation of our Phase 1 HNV study is a significant milestone for PepGen and marks our transition to a clinical stage company. We look forward to reporting topline data from this trial by the end of 2022 and expect that this data will enable our future studies in DMD patients to initiate in early 2023.”
Click the button below to read the full press release. We will continue to follow the progress of this Phase 1 study and keep you updated.