Lonvoguran Ziclumeran - In Vivo CRISPR Gene Editing in Hereditary Angioedema.

A single intravenous infusion of lonvoguran ziclumeran reduces hereditary angioedema attack rates by 87% compared to placebo in a phase 3 trial.

• Why it matters: Hereditary angioedema is a rare, life-threatening condition with recurrent swelling attacks, and current treatments may be insufficient or require multiple doses, highlighting the need for effective single-dose therapies.
• What they did: Researchers conducted a double-blind, randomized phase 3 trial involving 80 patients aged 16 or older, comparing a 50 mg dose of lonvo-z to placebo, measuring attack rates from week 5 to 28.
• The result: The median attack rate was 0.26 in the lonvo-z group versus 2.10 in the placebo group, with no serious adverse events reported, suggesting lonvo-z may offer a significant reduction in attack frequency.