Wave Life Sciences announces upcoming presentations at MDA conference

Wave Life Sciences announced its upcoming presentations at the 2024 Muscular Dystrophy Association, MDA, Clinical & Scientific Conference, taking place March 3-6 in Orlando, FL. Wave’s poster presentations will highlight the best-in-class potential of WVE-N531 in Duchenne muscular dystrophy, which is currently being evaluated in the Phase 2, potentially registrational FORWARD-53 study. Highlights from the presentations include: Data from the Phase 1b/2 proof-of-concept study of WVE-N531 in boys with DMD amenable to exon 53 skipping, which demonstrate uptake of WVE-N531 in satellite cells of all participants in the study. Preclinical data for WVE-N531 in non-human primates, which demonstrate that Wave’s PN chemistry significantly enhanced drug concentrations in skeletal muscle, with even higher exposure in the heart and diaphragm. These data suggest that WVE-N531 muscle concentrations in the clinic may be higher in heart and diaphragm than in skeletal muscle. Preclinical data for Wave’s exon skipping programs beyond exon 53, which reinforce the impact of PN chemistry for enabling high tissue concentrations, exon skipping and dystrophin restoration in preclinical models.