Intellia Therapeutics Advances In Vivo CRISPR Therapy Lonvo-z to FDA After Positive Phase 3 HAELO Trial Results

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Intellia Therapeutics' Phase 3 HAELO trial for lonvoguran ziclumeran (lonvo-z) met its primary endpoint, reducing HAE swelling attacks by 87% compared to placebo over six months in 80 patients.123

62% of lonvo-z patients were attack- and therapy-free during the six-month period, versus 11% on placebo; all key secondary endpoints met.123

The therapy showed a favorable safety profile with mild or moderate adverse events like infusion reactions, headache, and fatigue; no serious adverse events in the lonvo-z arm.123

Intellia initiated a rolling BLA submission to the FDA, aiming to complete it in the second half of 2026 for potential U.S. launch in H1 2027.123

HAELO marks the first positive Phase 3 results for an in vivo CRISPR-based gene editing therapy.234

Sources:

1. https://www.fiercebiotech.com/biotech/intellia-races-vivo-crispr-therapy-fda-after-phase-3-data-paint-compelling-picture

2. https://www.biospace.com/drug-development/intellia-heads-to-fda-after-notching-first-late-stage-win-for-in-vivo-gene-editor

3. https://www.stocktitan.net/news/NTLA/intellia-therapeutics-reports-positive-phase-3-results-in-hereditary-tryqqz0yj7w3.html

4. https://markets.businessinsider.com/news/stocks/intellia-therapeutics-reports-positive-phase-3-results-in-hereditary-angioedema-marking-a-global-first-for-in-vivo-gene-editing-1036065906