Lonvoguran ziclumeran (lonvo-z) is the first in vivo CRISPR gene-editing therapy demonstrating robust clinical efficacy in hereditary angioedema (HAE) due to C1 inhibitor deficiency.
A single intravenous infusion of lonvo-z significantly lowered monthly HAE attack rates by 87% compared to placebo in a phase 3 randomized trial.
Lonvo-z displayed a manageable safety profile with mostly mild-to-moderate infusion-related adverse events and no serious or grade 3+ toxicities.
Complementary CRISPR therapies targeting kallikrein B1 (KLKB1), such as NTLA-2002, show congruent reductions in attack frequency and kallikrein protein levels, supporting gene-editing as a disease-modifying strategy.
