Intellia Therapeutics Presents Progress in Lead In Vivo Program at Cold Spring Harbor Laboratory’s Fourth Meeting on Genome Engineering: The CRISPR-Cas Revolution
Robust, dose-responsive liver editing and reduction of TTR protein shown
in humanized mouse model
Therapeutically relevant reduction of serum TTR protein correlating with liver editing
achieved in non-human primates after a single dose
“As a follow-up to our rodent and NHP data released previously, we shared data on the extensive preclinical characterization of our therapeutic candidates. Our leads, used in conjunction with our modular LNP delivery system, result in reductions in TTR protein levels in NHPs that, when achieved in humans, are associated with therapeutic benefit,” said Intellia President and Chief Executive Officer
Mice and NHP Data Results from Intellia’s ATTR Program
The data presented today demonstrated a robust and therapeutically relevant TTR gene knockout across multiple studies, including in a humanized mouse model, a conventional mouse model and NHPs:
- In one humanized mouse study, researchers found that editing rates were dose-responsive, and also observed liver editing up to ~80 percent across multiple lead guide candidates. In a second humanized mouse study, researchers observed a lack of amyloid deposits in relevant tissues, including the stomach, colon, sciatic nerve and dorsal root ganglion, and therefore, a potential reversal in disease.
- In the conventional mouse study, as previously reported in Cell Reports, researchers observed a durable, therapeutically meaningful and sustained knockdown of 97 percent in serum transthyretin protein levels, following 12 months post-LNP administration of CRISPR/Cas9 in wild-type mice.
- In initial NHP studies, NHPs administered with a single dose of LNP-derived CRISPR/Cas9, Intellia achieved a TTR protein knockdown of 60 to 80 percent, comparable to current therapeutic standards. The data built on results released as part of the company’s first quarter earnings announcement.
Collectively, these data included results from ongoing collaborations with researchers at
About Intellia’s Transthyretin Amyloidosis Program
Transthyretin amyloidosis (ATTR) is a systemic, debilitating and fatal disease caused by one of approximately 136 different inherited mutations in the TTR gene. Abnormal protein deposits caused by one of these genetic mutations may affect both the peripheral and autonomic nervous systems, resulting in a variety of symptoms that develop in people as early as age 20. (Sources:
Intellia’s initial in vivo programs focus on the use of lipid nanoparticles (LNPs) for delivery of CRISPR/Cas9 components to the liver. The company’s lead in vivo program targets ATTR and is being co-developed with Regeneron Pharmaceuticals, Inc. Intellia aims to achieve knockout editing of specific DNA in mutated TTR genes within hepatocytes that cause damaging transthyretin protein deposits in heart, nerves and other tissues in the body. Intellia anticipates submitting an Investigational New Drug application for ATTR by the end of 2019.
This press release contains “forward-looking statements” of
Any forward-looking statements in this presentation are based on management’s current expectations and beliefs of future events, and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to: risks related to Intellia’s ability to protect and maintain our intellectual property position; risks related to the ability of our licensors to protect and maintain their intellectual property position; uncertainties related to the initiation and conduct of studies and other development requirements for our product candidates; the risk that any one or more of Intellia’s product candidates will not be successfully developed and commercialized; the risk that the results of preclinical studies will not be predictive of future results in connection with future studies; and the risk that Intellia’s collaborations with Novartis or Regeneron or its other ex vivo collaborations will not continue or will not be successful. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause Intellia’s actual results to differ from those contained in the forward-looking statements, see the section entitled “Risk Factors” in Intellia’s most recent annual report on Form 10-K and quarterly reports on Form 10-Q filed with the Securities and Exchange Commission, as well as discussions of potential risks, uncertainties, and other important factors in Intellia’s other filings with the Securities and Exchange Commission. All information in this presentation is as of the date of the release, and Intellia Therapeutics undertakes no duty to update this information unless required by law.
Senior Vice President
External Affairs & Communications
External Affairs & Communications
Source: Intellia Therapeutics, Inc.