Intellia Therapeutics, Inc.
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- Intellia Therapeutics, Inc. Intellia Therapeutics, Inc. (NTLA) is a clinical-stage biotechnology company focused on developing potentially curative CRISPR/Cas9-based gene editing therapeutics for severe genetic diseases, cancers, and autoimmune disorders; its core pipeline includes nexiguran ziclumeran (nex-z, formerly NTLA-2001) in Phase 3 trials for transthyretin (ATTR) amyloidosis with polyneuropathy (ATTRv-PN) and cardiomyopathy (ATTR-CM), lonvoguran ziclumeran (lonvo-z, formerly NTLA-2002) in Phase 3 HAELO trial for hereditary angioedema (HAE) with topline data expected mid-2026, NTLA-3001 for ATTR amyloidosis, NTLA-4001 for T-cell malignancies, and research programs in hemophilia B (partnered with Regeneron Pharmaceuticals, Inc.), hematological disorders such as sickle cell disease and beta-thalassemia, as well as immuno-oncology and autoimmune disease targets. Founded in 2014 and headquartered in Cambridge, Massachusetts, the company leverages a modular gene editing platform for in vivo and ex vivo therapies, supported by collaborations including Regeneron for ATTR amyloidosis co-development and cost reimbursements. Recent developments encompass completion of enrollment in the Phase 3 HAELO trial for lonvo-z in September 2025 ahead of schedule, presentation of positive longer-term Phase 1/2 data for nex-z and lonvo-z at ACAAI 2025 and other conferences, advancement in the Phase 3 MAGNITUDE trial for ATTR-CM, a strategic reorganization in early 2025 to prioritize late-stage programs, inducement equity grants under the 2024 Inducement Plan to support expansion, and a cash position of approximately $670 million as of Q3 2025 expected to fund operations into mid-2027 including potential U.S. commercial launch of lonvo-z in H1 2027.
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