Intellia heads to FDA with first in vivo CRISPR-based gene editing therapy

The most advanced clinical trial for an in vivo gene editor, lonvoguran ziclumeran (lonvo-z) reported positive phase 3 results in hereditary angioedema. Intellia Therapeutics announced that its systemic CRISPR–Cas9 gene editing medicine had met primary and secondary endpoints, leading the biotech to start a rolling submission for US Food and Drug Administration approval.

The phase 3 HAELO trial of lonvo-z enrolled a total of 80 patients, with 52 on lonvo-z and 28 on placebo. A one-time dose reduced attacks by 87% versus placebo for six months, and 62% of patients were attack-free and off preventative therapy for that period.