by: Parent Project Muscular Dystrophy
This morning, Wave Life Sciences Ltd. announced the initiation of dosing in a Phase 1b/2a clinical trial expecting to enroll 15 boys, evaluating investigational WVE-N531 as a treatment for boys with Duchenne muscular dystrophy (DMD) who are amenable to exon 53 skipping. This phase of the trial will take place in Canada and the United Kingdom and with the possibility of expanding sites depending on the progress of the study.
WVE-N531 is Wave’s first exon skipping candidate, and the first candidate systemically administered by intravenous infusion, to utilize its novel PN backbone chemistry modifications.
PPMD is pleased to have our partners at Wave back in the Duchenne Community, as they pursue this potential therapeutic.
