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Investing.com -- Intellia Therapeutics Inc (NASDAQ:NTLA) stock soared 13.8% in premarket trading Monday after the gene editing company presented positive clinical data for its hereditary angioedema (HAE) treatment.
The Cambridge, Massachusetts-based company reported that 97% of patients who received a 50 mg dose of its lonvo-z treatment were both attack-free and long-term prophylaxis-free as of the data cutoff. Among the 32 patients who received this dose, 24 (75%) remained attack-free and LTP-free for at least seven months, with some maintaining this status for up to 32 months.
The data came from a pooled analysis of all patients who received the 50 mg dose in the company’s ongoing Phase 1/2 clinical trial. Notably, 10 of the 11 patients who originally received this dose in Phase 2 remained attack-free and LTP-free.
Intellia also reported that the treatment demonstrated a well-tolerated safety profile with up to three years of patient follow-up, with no new long-term risks identified. The most common treatment-emergent adverse events within 28 days of infusion were infusion-related reactions, fatigue, and headache.
The one-time treatment is being further evaluated in a global Phase 3 HAELO clinical trial that completed enrollment in September 2025, with topline results expected by mid-2026.
HAE is a rare genetic disorder characterized by unpredictable, painful swellings that typically require lifelong treatment. Lonvo-z uses CRISPR gene editing technology and is administered as a single intravenous infusion.
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