Novel in vivo CRISPR-based therapy lonvoguran ziclumeran (lonvo-z) dramatically lowered hereditary angioedema (HAE) attack frequency in the HAELO randomized trial.Lonvo-z resulted in 62% of patients being attack-free in the trial, as compared with 40-60% attack-free on long-term prophylaxis in the pivotal trials.If approved, lonvo-z could be the first in vivo CRISPR-cas gene editing therapy for any indication.
Novel in vivo CRISPR-based therapy lonvoguran ziclumeran (lonvo-z) dramatically lowered hereditary angioedema (HAE) attack frequency in the HAELO randomized trial.
HAE attacks dropped by a relative 87% after the single infusion, with 0.26 average monthly attacks from weeks 5 through 28 compared with 2.10 in the placebo group (P<0.001) while off long-term HAE prophylaxis, reported Danny Cohn, MD, PhD, of Amsterdam University Medical Center, and colleagues in the New England Journal of Medicine.
Over that same period, 62% of the patients who received lonvo-z were considered by the investigator to be HAE attack-free without the use of long-term prophylaxis, as compared with 11% in the placebo group, the researchers also reported at the European Academy of Allergy and Clinical Immunology meeting in Istanbul.
