NTLA-2002 (lonvoguran ziclumeran) is an investigational, single-dose in vivo CRISPR/Cas9 gene-editing therapy being developed by Intellia Therapeutics for the treatment of hereditary angioedema (HAE). NTLA-2002 is designed to inactivate the kallikrein B1 (KLKB1) gene, which encodes prekallikrein, the precursor of plasma kallikrein, a key driver of bradykinin-mediated angioedema attacks. The therapy is administered systemically as a single intravenous infusion of lipid nanoparticles (LNPs) that deliver a non-viral, two-component genome-editing system consisting of Cas9 messenger RNA and a guide RNA specific to KLKB1, enabling targeted gene disruption in hepatocytes. By reducing plasma kallikrein production, NTLA-2002 has the potential to provide continuous suppression of disease activity and prevent HAE attacks following a single treatment.
Disease: Hereditary Angioedema, HAE, (NCT06634420)
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Candidate summary:
Disease/Disease category:
- Immunodeficiencies
Official title:
HAELO: a Phase 3, Multinational, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of NTLA-2002 in Participants with Hereditary Angioedema (HAE)
Phases:
- Phase 3
Status:
Active not recruiting
Country:
- United States
- Australia
- Canada
- France
- Germany
- Netherlands
- New Zealand
- South Africa
- United Kingdom
