Intellia Therapeutics Announces Publication in Cell Reports of Preclinical Data Demonstrating Effective CRISPR/Cas9 Genome Ed...
28 February 2018 - 1:28AM
Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading genome editing
company developing curative therapeutics using CRISPR/Cas9
technology, announced that Cell Reports will publish at noon ET
today its manuscript, “A single administration of CRISPR/Cas9 lipid
nanoparticles achieves robust and persistent in vivo genome
editing.”
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 David Morrissey, Ph.D., senior
vice president, Platform and Delivery Technology, at Intellia. “Our
lipid nanoparticle system is a transient expression system that
enables CRISPR/Cas9 to make the intended gene edit and then clear
from the cells. Minimizing the duration of CRISPR/Cas9 components
in cells is desirable, as that may reduce the potential for safety
issues associated with the continued presence of those components.
The LNPs also allow us to re-dose, if needed, to attain the desired
target effect. This paper details the most effective systemic
delivery of CRISPR/Cas9 components reported to date, further
supporting our IND-enabling activities this year and future
potential treatments for liver-based genetic diseases.”
The data included in this publication build on earlier findings
initially released last year at the Le Stadium Conference on
Messenger RNA Therapeutics, and later presented at the 20th
Annual Meeting of the American Society of Gene and Cell Therapy and
the 13th Annual Meeting of the Oligonucleotide Therapeutics
Society.
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:
Amyloidosis Foundation and National Institutes of Health)
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 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. Non-human
primate studies are ongoing and are anticipated to lead to
IND-enabling activities in 2018.
About Intellia Therapeutics
Intellia Therapeutics is a leading genome editing company
focused on the development of proprietary curative therapeutics
using the CRISPR/Cas9 system. Intellia believes the CRISPR/Cas9
technology has the potential to transform medicine by permanently
editing disease-associated genes in the human body with a single
treatment course. Our combination of deep scientific, technical and
clinical development experience, along with our leading
intellectual property portfolio, puts us in a unique position to
unlock broad therapeutic applications of the CRISPR/Cas9 technology
and create a new class of therapeutic products. Learn more about
Intellia Therapeutics and CRISPR/Cas9 at intelliatx.com; follow us
on Twitter @intelliatweets.
Forward-Looking Statements
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
Securities and Exchange Commission, as well as discussions of
potential risks, uncertainties, and other important factors in our
other filings with the Securities and Exchange Commission.
All information and forward-looking statements in this press
release are as of the date of the release, and Intellia
Therapeutics undertakes no duty to update this information, whether
because of new information, future events or otherwise, unless
required by law.
Intellia Contacts:
Media: Jennifer Mound Smoter Senior Vice
President External Affairs & Communications +1 857-706-1071
jenn.smoter@intelliatx.com
Lynnea Olivarez Associate Director External Affairs &
Communications +1 956-330-1917 lynnea.olivarez@intelliatx.com
Investors: Lindsey Trickett Vice President
Investor Relations +1 857-285-6211
lindsey.trickett@intelliatx.com
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