Intellia's one-time CRISPR therapy for hereditary angioedema gets FDA priority review
Intellia Therapeutics said on 8 September 2026 that the US Food and Drug Administration has accepted for review its application for lonvo-z (lonvoguran ziclumeran) and granted it priority review, with a decision expected by 10 March 2027. The FDA has also indicated it does not currently plan to convene an expert advisory panel — a sign the agency sees no need for extra outside scrutiny.
Lonvo-z is a treatment for hereditary angioedema (HAE), a rare inherited disease that causes sudden, unpredictable and sometimes life-threatening swelling attacks in the face, throat, gut and limbs. Patients typically manage it with lifelong medicines taken regularly to prevent attacks. What makes lonvo-z different is that it is designed as a single, one-time dose: it uses CRISPR gene editing — a technology that makes precise changes to DNA inside the body — to switch off a gene in the liver that drives the swelling. If approved, Intellia says it would be the world's first in-body CRISPR-based therapy and the only one-time treatment for HAE, potentially freeing patients from taking ongoing medication.
The application is backed by Intellia's global Phase 3 HAELO trial, which enrolled 80 patients in just nine months and tested a single 50 mg dose in adults and adolescents aged 16 and older with Type 1 or Type 2 HAE.
