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.

Transthyretin (ATTR) Amyloidosis Program:

  • NTLA-2001 is an investigational therapy for the treatment of ATTR amyloidosis. It is the first CRISPR candidate to be administered systemically, or intravenously, to edit a gene inside the human body. 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 amyloidosis, 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.
  • 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 ATTR.
  • Interim Phase 1 clinical data presented in June and September 2022 demonstrated substantial, dose-dependent reduction of TTR protein, following a single dose of NTLA-2001 in people with ATTR amyloidosis with polyneuropathy or cardiomyopathy .
  • Intellia leads development and commercialization of NTLA-2001 as part of a multi-target discovery, development and commercialization collaboration with Regeneron.

Hereditary Angioedema (HAE) Program:

  • NTLA-2002 is Intellia’s wholly owned investigational therapy for the treatment of HAE and is our second in vivo knockout therapeutic candidate. NTLA-2002 is currently being studied in a first-in-human Phase 1/2 clinical trial. In September 2022, Intellia presented interim Phase 1/2 clinical data demonstrating deep, dose-dependent reductions in plasma kallikrein following a single dose of 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.

Alpha-1 Antitrypsin Deficiency Programs:

  • 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 AATD-associated lung disease and eliminate the need for sub-optimal weekly IV infusions of A1AT augmentation therapy or transplant in severe cases.
  • NTLA-2003 is designed to knockout the mutant SERPINA1 gene to potentially reduce and prevent accumulation of mutant A1AT protein. This development candidate aims to address AATD-associated liver disease and eliminate the need for liver 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.