WC Eend schreef op 6 februari 2025 18:58:
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Intellia Therapeutics Announces First Patient Dosed in the HAELO Phase 3 Study of NTLA-2002, an Investigational In Vivo CRISPR Gene Editing Treatment for Hereditary AngioedemaIntellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies, today announced the first patient has been dosed in the global Phase 3 study of NTLA-2002 for the treatment of hereditary angioedema (HAE). NTLA-2002 is a wholly owned investigational in vivo CRISPR-based therapy in development as a single-dose treatment for this potentially life-threatening disease. Intellia expects to complete enrollment in the second half of 2025 and submit a biologics license application (BLA) in 2026 to support the
Company’s plans for a U.S. launch in 2027.