CRISPR Therapeutics presented positive Phase 1b data for CTX310, its investigational in vivo gene-editing therapy, at the American Heart Association Scientific Sessions. The study evaluated a single-course treatment in patients with severe hypertriglyceridemia, a condition associated with increased risk of pancreatitis and cardiovascular disease.
At Day 30, a single infusion produced a mean 63% reduction in triglycerides. Circulating ANGPTL3, the target protein, fell by a mean of 65%. Non-HDL cholesterol fell by a mean of 51%.
CTX310 was reported to be well-tolerated, with no treatment-related serious adverse events observed.