Intellia Therapeutics (NASDAQ:NTLA) today announced positive topline results from its pivotal Phase 3 HAELO clinical trial evaluating lonvoguran ziclumeran (lonvo-z), a one-time in vivo CRISPR-based gene editing therapy for patients with hereditary angioedema (HAE).
The study met its primary and all key secondary endpoints with high statistical significance, potentially transforming the treatment landscape for this rare genetic disorder.
The trial, which enrolled 80 patients, demonstrated that a single 50 mg infusion of lonvo-z led to an 87% reduction in mean monthly HAE attacks compared to a placebo over a six-month efficacy period.
Patients in the treatment arm experienced a mean monthly attack rate of 0.26, versus 2.10 in the placebo group (p< 0.0001).
Furthermore, 62% of patients receiving the gene edit remained entirely free of both attacks and the need for conventional prophylactic therapy throughout the observation period.
Safety data remains favorable for the investigational therapy.
As of the February 10, 2026, data cutoff, no serious adverse events were reported in the lonvo-z treatment arm.
The therapy is designed to permanently reduce the production of plasma kallikrein, the protein responsible for the debilitating swelling episodes characteristic of HAE, by editing the KLKB1 gene directly within the liver.
Following these results, Intellia has initiated a rolling Biologics License Application (BLA) submission to the U.S. Food and Drug Administration.
If approved, the company anticipates a commercial launch in the first half of 2027.