Intellia Therapeutics, Inc. announced that interim results from the Phase 1 portion of the Phase 1/2 study of NTLA-2002 were published online in the New England Journal of Medicine (NEJM). NTLA-2002 is an investigational in vivo CRISPR-based gene editing therapy in development as a single-dose treatment for hereditary angioedema (HAE), a rare genetic condition that leads to potentially life-threatening swelling attacks. Additionally, the company remain on track to initiate a global pivotal study for NTLA-2002 in the second half of 2024, subject to regulatory feedback.

This marks the second consecutive Intellia in vivo CRISPR- based program to have its initial clinical data published in the New England Journal of medicine, further supporting the immense potential impact its proprietary gene editing platform could have on the future of human health. The reported data showed that a single dose of NTLA-2002 led to a 95% mean reduction in monthly HAE attack rate across all 10 patients in the Phase 1 portion. Intellia's ongoing Phase 1/2 study is evaluating the safety and activity of NTLA-2002 in adults with Type I or Type II hereditary angioedema (HAE).

Interim Phase 1 clinical data showed dramatic reductions in attack rate, as well as consistent, deep and durable reductions in kallikrein levels. NTLA-2002 has received five notable regulatory designations, including Orphan Drug and RMAT Designation by the U.S. Food and Drug Administration, the Innovation Passport by the U.K. Medicines and Healthcare products Regulatory Agency (MHRA), Priority Medicines (PRIME) Designation by the European Medicines Agency, as well as Orphan Drug Designation by the European Commission. These forward-looking statements include, but are not limited to: risks related to Intellia's relationship with third parties, including its licensors and licensees; risks related to the ability of its licensors to protect and maintain their intellectual property position; uncertainties related to the authorization, initiation, enrollment and conduct of studies and other development requirements for its product candidates, including NTLA-2002; the risk that NTLA-2002 will not be successfully developed and commercialized; and the risk that the results of preclinical stu dies or clinical studies, such as the clinical study of NTLA-2002, will not be predictive of future results in connection with future studies for the same product candidate or Intellia's other product candidates.

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