Lonvoguran Ziclumeran - In Vivo CRISPR Gene Editing in Hereditary Angioedema

The authors aimed to evaluate the efficacy and safety of lonvoguran ziclumeran (lonvo-z), an investigational CRISPR-based gene-editing treatment, in reducing the frequency of attacks in patients with hereditary angioedema due to C1 inhibitor deficiency. In a phase 3 trial, a single intravenous infusion of lonvo-z significantly decreased the monthly attack rate compared to placebo, with a relative reduction of 87%. While adverse events were reported in both groups, no serious complications occurred in the lonvo-z group, indicating a favorable safety profile.