Intellia Therapeutics Reports Positive Phase 3 Results in Hereditary Angioedema, Marking a Global First for In Vivo Gene Editing
Intellia Therapeutics • • general
Key Points:
- Intellia Therapeutics announced that its Phase 3 HAELO trial of lonvoguran ziclumeran (lonvo-z), a one-time in vivo CRISPR gene editing treatment for hereditary angioedema (HAE), met its primary and all key secondary endpoints with favorable safety and tolerability results.
- A single dose of lonvo-z reduced HAE attacks by 87% compared to placebo over six months, with 62% of treated patients being attack- and therapy-free during this period, demonstrating the potential to eliminate the need for ongoing prophylactic treatment.
- Intellia has initiated a rolling biologics license application (BLA) submission with the FDA and anticipates a potential U.S. launch of lonvo-z in the first half of 2027, pending regulatory approval.
- The trial enrolled 80 patients, including those on long-term prophylaxis who discontinued prior therapies before dosing; no serious adverse events were observed, and all adverse events were mild or moderate.
- Additional clinical data will be presented at the 2026 European Academy of Allergy and Clinical Immunology Congress, and Intellia will host a webcast to discuss the topline results.
