lonvoguran

Lonvoguran (NTLA-2002) is an investigational, *in vivo* CRISPR/Cas9 genome editing therapy developed by Intellia Therapeutics for the treatment of hereditary angioedema (HAE). It is designed to permanently reduce the production of plasma kallikrein by specifically inactivating the *KLKB1* (Kallikrein B1) gene in hepatocytes. By knocking out this gene, the therapy lowers levels of prekallikrein, which in turn prevents the excessive formation of bradykinin—the primary mediator of the painful and potentially life-threatening swelling attacks characteristic of HAE. Lonvoguran is delivered via lipid nanoparticles (LNPs) through a single intravenous infusion, aiming to provide a functional cure and eliminate the need for chronic prophylactic treatments.

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lonvoguran