Intellia Therapeutics Advances in Hereditary Angioedema Treatment with Durable One-Dose CRISPR Gene Editing Therapy

1. Positive Long-Term Data: Intellia Therapeutics has announced positive long-term data from its ongoing Phase 1 study of NTLA-2002, an investigational in vivo CRISPR-based gene editing therapy for hereditary angioedema (HAE).
2. Durability of One-Dose Option: The data show that a single dose of NTLA-2002 led to a 98% mean reduction in monthly HAE attack rates, with an average follow-up of over 20 months across all patients.
3. Attack-Free Patients: Eight out of 10 patients remain completely attack-free following the 16-week primary observation period through the latest follow-up, including patients with the most severe disease.
4. Favorable Safety Profile: NTLA-2002 demonstrated a favorable safety and tolerability profile at all dose levels, with no dose-limiting toxicities, serious adverse events, or adverse events of Grade 3 or higher observed.
5. Clinical Development Plans: Intellia plans to initiate a pivotal Phase 3 trial of NTLA-2002 in the second half of 2024, subject to regulatory feedback, following the completion of the Phase 2 study.
6. Investor Webcast: Intellia hosted a live webcast on June 3, 2024, to discuss the long-term data and provide updates on the clinical development program for NTLA-2002.