Intellia Therapeutics Inc. (NASDAQ: NTLA) has initiated a rolling submission of a biologics license application to the U.S. Food and Drug Administration for lonvoguran ziclumeran, its gene editing treatment for hereditary angioedema.

The Cambridge, Massachusetts-based company announced the regulatory filing alongside positive results from its Phase 3 HAELO clinical trial. The trial met its primary and key secondary endpoints, with patients receiving a single dose of the treatment experiencing freedom from hereditary angioedema attacks and ongoing therapy use during a six-month observation period.

Lonvoguran ziclumeran, formerly known as NTLA-2002, uses CRISPR gene editing technology to inactivate the kallikrein B1 gene, intended to permanently lower kallikrein and bradykinin levels. The treatment is designed as a one-time outpatient administration.

The FDA previously granted the therapy Regenerative Medicine Advanced Therapy designation for hereditary angioedema treatment, which allows for the rolling submission process. This designation enables companies to submit portions of their application over time and provides expedited FDA review.

Intellia expects to complete its biologics license application submission in the second half of 2026. If accepted, the FDA will determine whether to grant priority review and provide a target action date. The company plans a commercial launch in the first half of 2027 if approved.

Hereditary angioedema affects approximately one in 50,000 people and causes severe, recurring inflammatory attacks in various organs and tissues. Current treatments typically require lifelong therapies with frequent intravenous or subcutaneous administration.

The therapy has received five regulatory designations, including Orphan Drug designation from both the FDA and European Commission, and Priority Medicines designation from the European Medicines Agency.