NTLA-2002 HAELO Phase 3 Enrollment Complete

NTLA-2002 is an in vivo CRISPR gene-editing therapy developed by Intellia Therapeutics for hereditary angioedema (HAE) — a rare genetic condition marked by recurrent, unpredictable attacks of severe swelling in the skin, gut, and airway that can be life-threatening. A single intravenous infusion delivers CRISPR/Cas9 via lipid nanoparticles to liver cells, permanently knocking out the KLKB1 gene to reduce plasma kallikrein — the enzyme whose overactivity triggers HAE attacks — providing durable protection without ongoing medication. NTLA-2002 is in a pivotal Phase 3 trial and is the first wholly in-vivo CRISPR therapy in late-stage testing.