Wave claims early lead in second-generation exon 53 skipping

Wave Life Sciences is taking another step forward in its comeback story with Phase II data that put WVE-N531 in a leading position among next-generation exon 53 skipping antisense therapies for Duchenne muscular dystrophy.

In the FORWARD-53 study, a 10 mg/kg dose administered twice-weekly led to a mean absolute muscle content-adjusted dystrophin expression of 9.0%, and a mean unadjusted dystrophin expression of 5.5% of normal across the 11 patients treated. Mean exon skipping was 57% based on RT-PCR measurements...