Intellia’s in vivo CRISPR therapy lonvo-z (lonvoguran ziclumeran) achieved major efficacy in the Phase 3 HAELO study for hereditary angioedema, cutting swelling attack rates by 87% versus placebo at six months and placing the program on a potential path to regulatory submission. The company has already initiated a rolling submission with the FDA. The Phase 3 dataset also reported that more than 60% of patients became attack-free during the study period with no serious safety signals reported in the highlighted coverage. Intellia’s approach edits genes in patients’ bodies rather than relying on ex vivo edited cells. Market interpretation has focused on whether one-time dosing can outweigh concerns around durability and the permanence of in vivo editing, while the FDA review timeline becomes a key near-term gating item.