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Wave Life Sciences: Positive Update to Ongoing Phase 1b/2a FOCUS-C9 Study

April 11, 2022

Defeat Duchenne Canada is pleased to share the news that Wave Life Sciences is advancing three Phase 1b/2a clinical trials with its next-generation PN chemistry-containing investigational candidates in C9orf72-associated amyotrophic lateral sclerosis (C9-ALS) and frontotemporal dementia (C9-FTD), Huntington’s disease and Duchenne muscular dystrophy.

WVE-004 in C9-ALS/FTD is being investigated in the FOCUS-C9 trial (NCT04931862) and WVE-003 in Huntington’s disease (HD), is being investigated in the SELECT-HD trial (NCT05032196). Additionally, Wave is investigating WVE-N531 targeting exon 53 in Duchenne muscular dystrophy (DMD) (NCT04906460). Wave expects to share clinical data later in 2022 for WVE-N531, in addition to WVE-004 and WVE-003, to provide further insight into the clinical effects of PN chemistry and enable decision-making for these programs.

We will keep you updated on any further developments. Click the button below to read the full press release.