Intellia Therapeutics Announces Progress with CRISPR/Cas9 at the American Society of Gene & Cell Therapy Annual Meeting
May 13 2017 - 7:40AM
Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading genome editing
company focused on the development of potentially curative
therapeutics using CRISPR technology, presented an update on its
long-term mouse genome editing and delivery studies and shared new,
first-time data in rat models demonstrating consistent
dose-dependent editing, at the American Society of Gene & Cell
Therapy’s Annual Meeting (ASGCT).
These data, featured in a platform presentation on
Saturday, May 13 at ASGCT showed:
- Six-month mouse study data demonstrating both durability and
high editing efficiency in vivo, with approximately 70 percent
editing at the target DNA site with a single intravenous dose. A 97
percent reduction of serum transthyretin (TTR) protein levels also
was sustained.
- In addition, robust, dose-responsive lipid nanoparticle
(LNP)-mediated editing of the TTR gene in rat livers following
single intravenous administration; up to 66 percent editing at the
target DNA site and up to 91 percent reduction in serum TTR protein
levels.
- Both studies were conducted using Intellia’s proprietary LNP
delivery system, providing high levels of liver delivery and rapid
clearance.
“Data from the additional rat study further
validates the in vivo CRISPR/Cas9 platform using Intellia’s
proprietary LNP delivery system,” said David Morrissey, Ph.D.,
senior vice president, Platform and Delivery Technology. “In both
species, we saw unprecedented in vivo liver editing results and
consistent delivery of CRISPR/Cas9 with systemic administration
using LNPs, while also showing the ability to expand our studies in
larger species.”
About Intellia Therapeutics
Intellia Therapeutics is a leading genome editing
company focused on the development of proprietary, potentially
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" of Intellia within the meaning of the Private
Securities Litigation Reform Act of 1995. These forward-looking
statements include, but are not limited to, express or implied
statements regarding Intellia’s ability to advance and expand the
CRISPR/Cas9 technology to develop into human therapeutic products;
our ability to achieve stable liver editing; effective genome
editing with a single treatment dose; and the potential timing and
advancement of our preclinical studies and clinical trials. Any
forward-looking statements in this press release 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 be predictive of future results
in connection with future studies; and the risk that Intellia’s
collaborations with Novartis or Regeneron 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 filed
with the Securities and Exchange Commission, as well as discussions
of potential risks, uncertainties, and other important factors in
Intellia’s subsequent filings with the Securities and Exchange
Commission. All information in this press release is as of the date
of the release, and Intellia Therapeutics undertakes no duty to
update this information unless required by law.
Intellia Contacts:
Media Contact:
Jennifer Mound Smoter
Senior Vice President, External Affairs & Communications
+1 857-706-1071
jenn.smoter@intelliatx.com
Investor Contact:
Graeme Bell
Executive Vice President, Chief Financial Officer
+1 857-706-1081
graeme.bell@intelliatx.com
Intellia Therapeutics (NASDAQ:NTLA)
Historical Stock Chart
From Jun 2024 to Jul 2024
Intellia Therapeutics (NASDAQ:NTLA)
Historical Stock Chart
From Jul 2023 to Jul 2024