Biotechnology company focused on developing CRISPR/Cas9-based therapies for genetic diseases.
Intellia Therapeutics, Inc., a genome editing company, is dedicated to the development of transformative therapeutics using its proprietary CRISPR/Cas9 technology. Among its in vivo programs, NTLA-2001 is in Phase 1 clinical trials for treating transthyretin amyloidosis, while NTLA-2002 targets hereditary angioedema. The company's liver-focused programs also include treatments for hemophilia A and B, hyperoxaluria Type 1, and alpha-1 antitrypsin deficiency.
Intellia's ex vivo pipeline features NTLA-5001, designed for treating acute myeloid leukemia, alongside various proprietary programs aimed at developing engineered cell therapies for oncology and autoimmune disorders. Leveraging the powerful CRISPR/Cas9 system, Intellia's innovative approach to gene editing underpins its broad therapeutic pipeline.
The company has established multiple strategic collaborations to enhance its development efforts. These include a partnership with Novartis Institutes for BioMedical Research, Inc. for engineering hematopoietic stem cells to treat sickle cell disease, and a co-development agreement with Regeneron Pharmaceuticals, Inc. for potential treatments for hemophilia A and B. Additionally, Intellia collaborates with Ospedale San Raffaele and SparingVision SAS to develop novel genomic medicines for ocular diseases.
Originally known as AZRN, Inc., Intellia Therapeutics, Inc. was incorporated in 2014 and is headquartered in Cambridge, Massachusetts. The company continues to leverage its cutting-edge genome editing technology to create innovative treatments for serious diseases.