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Intellia Therapeutics Reports Positive Phase 3 Results For HAE Gene Therapy; Stock Up

By RTTNews Staff Writer   ✉  | Published:  | Google News Follow Us  | Join Us

Intellia Therapeutics, Inc. (NTLA) on Monday reported positive topline results from its Phase 3 HAELO trial of lonvo-z, a CRISPR-based gene editing therapy for Hereditary angioedema (HAE). The study met its primary goal.

HAE is a rare genetic condition in which patients experience
recurrent and potentially life-threatening swelling attacks in
various parts of their body.

Results showed a one-time infusion of lonvo-z cut attack rates by 87% versus placebo over six months, with monthly attacks averaging 0.26 compared with 2.10 in the placebo group. The company said a significantly higher proportion of patients remained attack-free during the study period versus placebo.

Lonvo-z demonstrated a favorable safety profile, with all reported adverse events being mild to moderate.

Intellia said it has begun a rolling biologics license application submission with the U.S. Food and Drug Administration and expects a potential U.S. launch in the first half of 2027, if approved.

Shares rose more than 4% in pre-market trading after closing at $13.63 on Friday.

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