Intellia Therapeutics Announces Publication in Cell Reports of Preclinical Data Demonstrating Effective CRISPR/Cas9 Genome Editing Using Lipid Nanoparticle (LNP) Delivery Technology
- Intellia researchers achieved 97 percent reduction in serum transthyretin protein levels in mice through a single administration, with a durability of at least 12 months
- CRISPR/Cas9 administered via LNP was well-tolerated with no observed adverse events
- CRISPR/Cas9 components shown to be undetectable three days after administration
The lipid nanoparticle (LNP) delivery of Cas9 mRNA and sgRNA resulted in 97 percent reduction in mouse transthyretin (TTR) protein levels in the liver, and the reduction was sustained for at least 12 months. The publication also documents that CRISPR/Cas9 components were undetectable in mice within three days after administration of Intellia’s LNP delivery system. Researchers further demonstrated that Intellia’s LNP technology is a similarly robust and effective delivery method for CRISPR/Cas9-mediated knockdown in rats, a higher rodent species.
“These data show that our proprietary lipid nanoparticle technology achieves significant and enduring editing of the TTR gene through a single dose,” said
The data included in this publication build on earlier findings initially released last year at the
About Intellia’s transthyretin amyloidosis (ATTR) program
Transthyretin amyloidosis (ATTR) is a slowly progressive and debilitating 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 sentinel 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
This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statement in this press release include, but are not limited to, express or implied statements regarding the successful development of CRISPR/Cas9-based therapies to treat diseases, including transthyretin amyloidosis (ATTR); the translation of the results from animal models to human therapies; and the Company’s or its collaborator’s ability and intention to develop, seek regulatory approval for, and commercialize therapies to treat disease using CRISPR/Cas9. Any forward-looking statements in this press release are uncertain, based on management's current expectations of future events, occurrences, actions and plans, and subject to various 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. The Company may not actually execute or obtain the results from the plans, goals, efforts or opportunities disclosed in these forward-looking statements, and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, goals, efforts or opportunities disclosed in these forward-looking statements as a result of various factors including: uncertainties inherent in the implementation and execution of preclinical studies and clinical trials, and preclinical and clinical development of the Company’s or its collaborator’s products candidates; availability and timing of results from preclinical studies and clinical trials; whether interim results from a preclinical trial will be predictive of the final results of the preclinical or clinical trials or the results of future trials; expectations and requirements for regulatory approvals to conduct trials or to market products; and availability of funding sufficient for the Company’s or its collaborator’s foreseeable and unforeseeable operating expenses and capital expenditure requirements. For a discussion of risks and uncertainties, and other important factors, any of which could cause our actual results to differ from those contained in the forward-looking statements, see the section entitled "Risk Factors" in our most recent quarterly report on Form 10-Q filed with the
All information and forward-looking statements in this press release are as of the date of the release, and
Senior Vice President
Source: Intellia Therapeutics, Inc.