NTLA-2002
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.
Upcoming catalysts
Programs
Hereditary Angioedema
Hereditary angioedema causes unpredictable attacks of severe swelling in the skin, gut, and airway — laryngeal attacks can be fatal — driven by uncontrolled bradykinin from C1 inhibitor deficiency; current subcutaneous prophylactics require ongoing injections every 2–4 weeks indefinitely. NTLA-2002 aims to eliminate attacks with a single IV infusion by permanently knocking out KLKB1 in the liver; Phase 3 HAELO trial enrollment completion in 2026 anchors the 2027 topline readout.