We’re advancing our first investigational genome editing medicine into the clinic in 2020.
Intellia believes the versatility of our modular approach enables us to maximize the power and versatility of CRISPR/Cas9 and, importantly, provides us with options to rapidly pursue treatments for numerous diseases with limited treatment options available.
NTLA-2001 is our first program where we intend to substantively reduce the TTR protein causing the ATTR disease. Our goal is to treat patients with ATTR regardless if they have the hereditary or wild-type form of the disease.
Our in vivo pipeline includes a proprietary program targeting transthyretin amyloidosis (ATTR) for the treatment of polyneuropathy and cardiomyopathy, which we are co-developing with Regeneron Pharmaceuticals, Inc.
ATTR Program:
- 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. In addition, Intellia completed a 12-month durability study of its lead lipid nanoparticle (LNP) formulation in support of NTLA-2001, maintaining an average reduction of >95% of transthyretin (TTR) protein and sustained liver editing after a single dose in non-human primates (NHPs). NTLA-2001 is part of a co-development/co-promotion (Co/Co) agreement with Regeneron Pharmaceuticals, Inc. (Regeneron), with Intellia as the lead development and commercialization party.
Plan to nominate DC for HAE in 1H 2020:
- Hereditary angioedema (HAE) is Intellia’s second in vivo knockout development program. 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. Most patients with HAE have a C1 esterase inhibitor (C1-INH) protein deficiency, which normally prevents the unregulated release and buildup of bradykinin. Using its modular LNP-based CRISPR/Cas9 delivery system, Intellia aims to knock out the kallikrein B1 (KLKB1) gene, which is part of a biological pathway that activates bradykinin. Knocking out this gene should reduce the undesired bradykinin activity in HAE patients.
- 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.
To keep up with our progress, check out our pipeline.