Intellia rallies on Phase 1 data for CRISPR-based HAE therapy
Intellia Therapeutics stock jumped 13% Monday in the wake of positive long-term data from a Phase 1 study of its CRISPR-based gene therapy NTLA-2002 in the treatment of hereditary angioedema, or HAE. The biotech company said in a statement that a single dose of NTLA-2002 led to a 98% mean reduction in the monthly attack rate, with an average follow-up of over 20 months across all patients. It added that eight out of ten patients were completely attack-free following a 16-week primary observation period through the last follow-up, including patients with the most severe disease. Intellia presented the data over the weekend at the European Academy of Allergy and Clinical Immunology Congress 2024 meeting in Spain. The company expects to report topline Phase 2 results for the therapy mid-year. A pivotal Phase 3 study is expected to start in the second half of 2024, subject to regulatory feedback.