Wave Life Sciences recently shared that the company has initiated dosing and has fully enrolled the Phase 2 FORWARD-53 trial of investigational WVE-N531, a potential exon skipping therapy targeting people with Duchenne amenable to exon 53 skipping. 

FORWARD-53 is a potentially registrational, open-label Phase 2 clinical trial that has enrolled 10 participants with Duchenne who are amenable to exon 53 skipping. The primary endpoint is functional dystrophin protein levels which will be measured following 24 and 48 weeks of every other week dosing at 10 mg/kg. Wave also indicated that the trial is also evaluating safety and tolerability, digital and functional endpoints.

PPMD is pleased to learn this news and we look forward to more updates from Wave as their research into this potential therapeutic continues.

Read Wave’s community letter here.