Intellia Therapeutics Reports Promising Long-Term Data for Single-Dose HAE 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 gene editing treatment for hereditary angioedema (HAE).
2. Single-Dose Efficacy: The data show that a single dose of NTLA-2002 led to a 98% mean reduction in monthly HAE attack rate, with an average follow-up of over 20 months across all patients.
3. Attack-Free Patients: Eight 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: The treatment has demonstrated a favorable safety and tolerability profile at all dose levels.
5. Potential Functional Cure: The data suggest that NTLA-2002 could be a one-time, potential functional cure for HAE, a rare genetic condition characterized by severe, recurring, and unpredictable inflammatory attacks.
6. Regulatory Designations: NTLA-2002 has received five notable regulatory designations, including Orphan Drug and RMAT Designation by the FDA.
7. Presentation at EAACI Congress: The updated data were presented at the EAACI Congress 2024, highlighting the potential of NTLA-2002 to revolutionize the treatment of HAE.