Our modular approach enables us to optimize the power and versatility of the CRISPR/Cas9 technology and, importantly, allows us to rapidly develop therapeutics for numerous diseases that currently have limited treatment options.

ATTR Program:

  • NTLA-2001 could be the first curative treatment for transthyretin (ATTR) amyloidosis. By applying the company’s in vivo liver knockout approach, NTLA-2001 has the potential for lifelong reduction of TTR protein and reversing disease progression with a single dose of treatment. The investigational therapy is delivered with Intellia’s proprietary non-viral lipid nanoparticle platform, which the company is using to develop other in vivo treatments. Our goal is to address all forms of ATTR, regardless of disease type, with a single dose of treatment.

  • ATTR is a progressive and fatal disease that results from the build-up of a misfolded form of the TTR protein, leading to diverse disease manifestations and disease progression, including peripheral neuropathy and cardiomyopathy.
  • NTLA-2001 is the first investigational CRISPR therapy candidate to be administered systemically, or intravenously, to edit genes inside the human body. Intellia’s proprietary non-viral platform deploys lipid nanoparticles to deliver to the liver a two-part genome editing system: guide RNA specific to the disease-causing gene and messenger RNA that encodes the Cas9 enzyme, which carries out the precision editing. Robust preclinical data, showing deep and long-lasting transthyretin (TTR) reduction following in vivo inactivation of the target gene, supports NTLA-2001’s potential as a single-administration therapeutic for transthyretin (ATTR) amyloidosis. Interim Phase 1 clinical data released in June 2021 demonstrated substantial, dose-dependent reduction of TTR protein following a single dose of NTLA-2001.
  • Intellia leads development and commercialization of NTLA-2001 as part of a multi-target discovery, development and commercialization collaboration with Regeneron.

HAE Program:

  • NTLA-2002 is Intellia’s wholly-owned development candidate for the treatment of hereditary angioedema (HAE) and is our second in vivo knockout therapeutic candidate. In December 2021, we announced that the first patient had been dosed with NTLA-2002.
  • HAE is a rare genetic disorder characterized by recurring and unpredictable severe swelling attacks in various parts of the body, and is significantly debilitating and disabling. The disease is caused by increased levels of bradykinin, a protein which leads to swelling. NTLA-2002 aims to prevent unregulated production of bradykinin by inactivating the kallikrein B1 (KLKB1) gene, which encodes for prekallikrein, the kallikrein precursor protein, through a single dose of treatment to ameliorate the frequency and intensity of these swelling attacks.

AATD-Lung Disease Program:

  • NTLA-3001 is Intellia’s first and wholly owned CRISPR/Cas9-mediated in vivo targeted gene insertion development candidate. It is designed with the aim to precisely insert a healthy copy of the SERPINA1 gene to potentially achieve steady, continuous expression of A1AT protein at therapeutic levels after a single dose. This approach aims to address alpha-1 antitrypsin deficiency (AATD)-associated lung disease and eliminate the need for sub-optimal weekly IV infusions of A1AT augmentation therapy or transplant in severe cases.

Hemophilia A and B Programs:

  • In June 2020, Intellia and Regeneron expanded and extended their collaboration to research and develop CRISPR/Cas9-based treatments. Under the terms of two co-development and co-commercialization agreements, Intellia and Regeneron agreed to co-develop potential hemophilia A and B CRISPR/Cas9-based treatments using their jointly owned targeted transgene insertion technology. Regeneron is the lead party for both hemophilia A and hemophilia B development programs.
  • These programs build on proprietary innovations developed by Intellia in its collaboration with Regeneron. Data presented in 2019 by Intellia highlighted the promise of Intellia’s technology by demonstrating the first CRISPR-mediated, targeted transgene insertion in the liver of non-human primates, which generated circulating human Factor IX, or FIX, protein at or above normal levels necessary to treat hemophilia B, a severe genetic bleeding disorder. View Press Release

To keep up with our progress, check out our pipeline.