Longevity & AgingSingle CRISPR Injection Cuts Hereditary Angioedema Attacks by 87% in Phase 3 Trial
Intellia Therapeutics has reported the world's first positive Phase 3 result for an in vivo CRISPR gene editing therapy. Their treatment, lonvo-z, targets hereditary angioedema — a rare condition causing life-threatening swelling attacks — with a single infusion. In the HAELO trial, patients saw an 87% reduction in monthly attacks versus placebo, and 62% remained completely attack-free over six months compared to just 11% on placebo. Quality-of-life scores improved by over 17 points, far exceeding the 6-point threshold considered clinically meaningful. The therapy works by permanently reducing production of kallikrein, the protein that triggers attacks, rather than requiring ongoing medication. Safety data were encouraging, with only mild-to-moderate side effects reported. Results were published in the New England Journal of Medicine.