Intellia Therapeutics, Inc.
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- Intellia - In Vivo Therapies
Intellia - In Vivo Therapies
The first patient was dosed with our investigational genome editing treatment for transthyretin (ATTR) amyloidosis in November 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.
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- 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 systemically delivered investigational CRISPR/Cas9 therapy to enter the clinic. Intellia dosed the first patient in the company’s global Phase 1 clinical trial in November 2020, evaluating NTLA-2001 for the treatment of hereditary transthyretin amyloidosis with polyneuropathy (ATTRv-PN). Once safety and an optimal dose have been determined, Intellia intends to further evaluate NTLA-2001 in a broader ATTR patient population. Beyond receiving authorization in the U.K. to initiate this trial, Intellia is submitting additional regulatory applications in other countries as part of its global clinical development plans.
- In support of NTLA-2001, Intellia completed a 12-month preclinical 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 wholly-owned development candidate for the treatment of hereditary angioedema (HAE) and is our second in vivo knockout therapeutic candidate. In June 2021, we submitted our first CTA to initiate a Phase 1 study and plan to enroll our first patient by the end of the year.
- 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 dose of treatment to ameliorate the frequency and intensity of these swelling attacks.
- 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