We’re advancing our first investigational genome editing medicine into the clinic in 2020.
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.
- NTLA-2001, our first proprietary in vivo development candidate, seeks to treat transthyretin amyloidosis (ATTR) by substantively reducing the transthyretin (TTR) protein production that causes the disease. Our goal is to address hereditary and wild-type ATTR, both polyneuropathy and cardiomyopathy, with a single course of treatment.
- ATTR is a progressive and fatal disease that results in systemic failure from the deposition of insoluble TTR protein fibrils into multiple organs, leading to diverse disease manifestations, including peripheral neuropathy and cardiomyopathy.
- NTLA-2001 is anticipated to be the first systemically delivered investigational CRISPR/Cas9 therapy to enter the clinic. Intellia announced in August 2020 that it submitted its first Clinical Trial Application (CTA) to the United Kingdom’s Medicines and Healthcare products Regulatory Agency (MHRA) to initiate a Phase 1 study of NTLA-2001 for the treatment of ATTR. Intellia is submitting additional regulatory applications to enable enrollment in other countries as part of its global clinical development plans.
- In support of NTLA-2001, Intellia completed a 12-month durability study of its lead lipid nanoparticle (LNP) formulation, which showed maintenance of an average reduction of >95% of serum TTR protein and sustained liver editing after a single dose in non-human primates.
- We lead the development of NTLA-2001, which we are co-developing with Regeneron.
- NTLA-2002 is Intellia’s development candidate for the treatment of hereditary angioedema (HAE) and is our second in vivo knockout therapeutic candidate. We expect to submit an IND or IND-equivalent in 2H 2021.
- 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 knocking out the prekallikrein B1 (KLKB1) gene through a single course of treatment to ameliorate the frequency and intensity of these swelling attacks.
- Intellia’s KLKB1 HAE program is subject to an option by Regeneron to enter into a co-development/co-promotion agreement, in which Intellia would remain the lead party.
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.