Single-Dose CRISPR Therapy Significantly Reduces Hereditary Angioedema Attacks in Phase 3 Trial
A single-dose CRISPR therapy, lonvoguran ziclumeran (lonvo-z or NTLA-2002), has demonstrated significant efficacy in reducing hereditary angioedema (HAE) attacks, according to results from the HAELO phase 3 randomized controlled trial. The study, conducted across 31 sites in nine countries, involved patients aged 16 and older with HAE due to C1 inhibitor deficiency. Participants received either a single 50-mg intravenous infusion of lonvo-z or a placebo. From week 5 through week 28, the mean monthly attack rate in the lonvo-z group was 0.26, compared to 2.10 in the placebo group, representing an 87% relative reduction. Furthermore, 62% of patients treated with lonvo-z remained attack-free, in contrast to 11% in the placebo group. The therapy also reduced attacks requiring on-demand treatment by 89% and moderate or severe attacks by 91%. While infusion reactions were common, all adverse events were mild (grade 1 or 2), and no serious adverse events occurred in the treatment group. Transient, asymptomatic li... 
