Intellia Therapeutics

Editing genetico in vivo per malattie genetiche rare e cardiovascolari.

Intellia Therapeutics, Inc. operates as a clinical-stage genome editing company focused on developing potentially curative therapeutics using CRISPR/Cas9-based technologies. The company offers clustered, regularly interspaced short palindromic repeats (“CRISPR”)/CRISPR associated 9 (“Cas9”) technology for genome editing. The company provides a modular platform, to advance in vivo and ex vivo therapies for diseases. The company's in vivo product candidates include nexiguran ziclumeran, or NTLA-2001 for the treatment of transthyretin amyloidosis; and NTLA-2002 for the treatment of hereditary angioedema. Additionally, it offers product candidates for the treatment of immuno-oncology and autoimmune diseases, and multiple in vivo programs to address diseases with significant unmet medical need by delivering gene editing therapeutics to organs outside the liver. The company has license and collaboration agreement with AvenCell Therapeutics, Inc. to develop allogeneic universal CAR-T cell therapies; Kyverna Therapeutics, Inc. for the development of an allogeneic CD19 CAR-T cell therapy for the treatment of various of B cell-mediated autoimmune diseases; ONK Therapeutics, Ltd. for the development of engineered NK cell therapies to cure patients with cancer; and ReCode Therapeutics, Inc. to develop novel genomic medicines for the treatment of cystic fibrosis. It also has collaboration agreements with Regeneron Pharmaceuticals, Inc., SparingVision SAS, and Rewrite Therapeutics Inc. The company was formerly known as AZRN, Inc. and changed its name to Intellia Therapeutics, Inc. in July 2014. Intellia Therapeutics, Inc. was incorporated in 2014 and is headquartered in Cambridge, Massachusetts.