Intellia Therapeutics, Inc. operates as a clinical-stage biopharmaceutical company leveraging CRISPR/Cas9 technology to permanently edit disease-causing genes. The company's pipeline is divided into in vivo programs, which use intravenously administered CRISPR to edit genes directly within specific target tissues, and ex vivo programs, which engineer human cells outside the body to treat cancer and autoimmune diseases. Key clinical candidates include nexiguran ziclumeran (nex-z) for transthyretin (ATTR) amyloidosis and lonvo-z for hereditary angioedema (HAE). Intellia collaborates with major pharmaceutical companies, including Regeneron and Novartis, to advance its research and commercialization efforts. Headquartered in Cambridge, Massachusetts, the company aims to provide single-dose, potentially lifelong cures for severe and life-threatening conditions.
