President and Chief Executive Officer, Intellia Therapeutics
Appears in 1 story
Led simultaneous Phase 3 data release and rolling BLA submission announcement on April 27, 2026
On April 27, 2026, Intellia Therapeutics reported that its Phase 3 HAELO trial of lonvoguran ziclumeran (lonvo-z) — a one-time, in-body CRISPR gene-editing therapy for hereditary angioedema (HAE) — met its primary endpoint and all key secondary endpoints. In the 80-patient, randomized, double-blind trial, a single intravenous infusion of lonvo-z cut the rate of debilitating swelling attacks by 87% compared with placebo in the primary observation window (weeks 5 through 28). Sixty-two percent of patients who received the therapy were completely attack-free and had stopped all preventive medications in that period. The safety profile was clean: all reported side effects were mild or moderate, with no serious adverse events, and infusion-related reactions, headache, and fatigue were the most common complaints.
Updated 14 hours ago
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