Intellia Therapeutics, Inc., a genome editing company, focuses on the development of therapeutics. The company's in vivo programs include NTLA-2001, which is in Phase 1 clinical trial for the treatment of transthyretin amyloidosis; and NTLA-2002 for the treatment of hereditary angioedema, as well as other liver-focused programs comprising hemophilia A and hemophilia B, hyperoxaluria Type 1, and alpha-1 antitrypsin deficiency. Its ex vivo pipeline includes NTLA-5001 for the treatment of acute myeloid leukemia; and proprietary programs focused on developing engineered cell therapies to treat various oncological and autoimmune disorders.
In addition, it offers tools comprising of Clustered, Regularly Interspaced Short Palindromic Repeats/CRISPR associated 9 (CRISPR/Cas9) system. Intellia Therapeutics, Inc. has license and collaboration agreements with Novartis Institutes for BioMedical Research, Inc. to engineer hematopoietic stem cells for the treatment of sickle cell disease; Regeneron Pharmaceuticals, Inc. to co-develop potential products for the treatment of hemophilia A and hemophilia B; Ospedale San Raffaele; and a strategic collaboration with SparingVision SAS to develop novel genomic medicines utilizing CRISPR/Cas9 technology for the treatment of ocular diseases. The company was formerly known as AZRN, Inc.
Intellia Therapeutics, Inc. was incorporated in 2014 and is headquartered in Cambridge, Massachusetts.