COMPANY RESEARCHIntellia Therapeutics is a U.S. biotechnology company developing medicines based on genome editing, including CRISPR-based technologies. Its research aims to create genetic interventions for serious diseases, with drug development programs evaluating targeted editing in relevant tissues. The company's therapeutic interests include rare inherited and protein-mediated conditions. Intellia is headquartered in Cambridge, Massachusetts, and its common stock trades on Nasdaq under NTLA. It operates as a research-driven therapeutic developer rather than a contract-service or diagnostic-only company. This entry summarizes enduring modality and disease-area characteristics without claiming that investigational therapies are approved.
Profile review: 2026-11-04 · Fields retain their individual source dates. Undisclosed values are not zero; early or historical events do not establish the latest financing. Awards and acquisitions are distinguished from equity rounds.
Intellia Therapeutics is a U.S. biotechnology company developing medicines based on genome editing, including CRISPR-based technologies. Its research aims to create genetic interventions for serious diseases, with drug development programs evaluating targeted editing in relevant tissues. The company's therapeutic interests include rare inherited and protein-mediated conditions. Intellia is headquartered in Cambridge, Massachusetts, and its common stock trades on Nasdaq under NTLA. It operates as a research-driven therapeutic developer rather than a contract-service or diagnostic-only company. This entry summarizes enduring modality and disease-area characteristics without claiming that investigational therapies are approved.
Nasdaq: NTLA
CRISPR gene editing · genome engineering
rare genetic diseases · hereditary angioedema · cardiomyopathy
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Disease tags match recorded text mentions and require program-level verification.
Intellia presents CRISPR gene-editing science, clinical programs, and scientific publications on its official website. Its gene-editing work belongs in this broader in vivo engineering field; it should not be labeled as equivalent to in vivo CAR-T.
Individual program detail is not recorded. Consult the original sources below.
Company-name matches are discovery leads; verify entity identity and transaction roles in the original source.
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Intellia's third-quarter update reports completed enrollment in the Phase 3 MAGNITUDE-2 trial of nexiguran ziclumeran for hereditary ATTR amyloidosis with polyneuropathy, exceeding the target of 60 randomized patients. The company also described commercial preparations for lonvoguran ziclumeran in hereditary angioedema, ahead of its March 10, 2027 FDA target action date. Approval remains pending.
Intellia announced FDA acceptance of its biologics license application for lonvoguran ziclumeran, or lonvo-z, in hereditary angioedema, with Priority Review and a March 10, 2027 target action date. The investigational treatment uses LNP-delivered CRISPR components for in vivo editing. Application acceptance advances the regulatory process but is not marketing approval and does not guarantee a favorable decision.
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