Dosing has begun in a clinical trial of WVE-004, Wave Life Sciences‘ investigational therapy for amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) associated with C9orf72 gene mutations. The Phase 1b/2a trial, called FOCUS-C9 (NCT04931862), is evaluating the safety and tolerability of single- and multiple-ascending doses of WVE-004 against a placebo in about 50 adults, ages 18 to 80, for six months (24 weeks). Recruitment is underway at a site in Utrecht, Netherlands, with a second site expected to soon open in Quebec, Canada. A site has also being approved for New Zealand which can hopefully start recruiting later this year. Click here to read more about the WVE-004 trial.
