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Intellia Therapeutics

Intellia Therapeutics

Clinical-stage biotechnology company

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Intellia releases first Phase 3 readout for an in-body CRISPR therapy

New Capabilities

Rolling BLA submission to FDA ongoing; ~$194.6M raised in April 2026 equity offering; cash runway into at least 2028; HAELO data to be presented at EAACI Congress June 12–15, 2026 in Istanbul. Commercial launch of lonvo-z targeted for H1 2027 if approved.

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