Intellia Therapeutics has had the Biologics License Application (BLA) for lonvoguran ziclumeran accepted by US health authorities with Priority Review status, with no advisory committee currently planned, positioning the asset to become the world's first approved in vivo CRISPR-based therapy and the only one-time treatment for hereditary angioedema (HAE). Lonvoguran ziclumeran uses CRISPR/Cas9 to permanently inactivate KLKB1 in an outpatient infusion, lowering kallikrein and preventing bradykinin-driven swelling attacks, and already holds US Orphan Drug and RMAT status, UK MHRA Innovation Passport, EMA PRIME, and EU Orphan Drug designation.

The BLA rests on the Phase III HAELO trial, fully enrolled at 80 patients in nine months, which showed an 87% reduction in mean monthly attacks versus placebo over weeks 5 to 28 (p<0.0001), with 62% of lonvoguran ziclumeran patients attack-free and prophylaxis-free over the six-month evaluation period versus 11% on placebo, and only mild or moderate adverse events such as infusion-related reactions, headache, fatigue, back pain, and upper respiratory tract infection.