Intellia Therapeutics Presents Progress in Lead In Vivo Program at Cold Spring Harbor Laboratory’s Fourth Meeting on Genome...
August 23 2018 - 4:01PM
Robust, dose-responsive
liver editing and reduction of TTR protein shown in humanized
mouse model
Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading genome editing
company focused on developing curative therapeutics using
CRISPR/Cas9 technology, presented data from studies demonstrating a
decrease in amyloid deposition in multiple tissues of a humanized
mouse model of hereditary transthyretin amyloidosis (ATTR) after a
single dose of lipid nanoparticles (LNPs). The company also
presented non-human primate (NHP) data from initial studies showing
a therapeutically meaningful level of TTR protein reduction that
correlated with robust and significant liver editing following a
single administration of LNPs. Yong Chang, Ph.D., vice president,
safety pharmacology, Intellia, presented these data today, at Cold
Spring Harbor Laboratory’s (CSHL) fourth meeting on Genome
Engineering: The CRISPR-Cas Revolution, in Cold Spring Harbor, N.Y.
“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 John Leonard, M.D. “Now, with IND-enabling activities for
our lead in vivo program, ATTR, underway, we are one step closer to
realizing the potential of CRISPR/Cas9 genome editing for
developing curative treatments for chronic genetic diseases like
ATTR. These additional activities are focused on confirming these
initial results and identifying appropriate dose ranges for the
development of potential human therapeutics.”
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 Regeneron Pharmaceuticals, Inc.,
and the University of Porto in Portugal, where ATTR is endemic in
certain regions. Click here to view the full presentation,
entitled, “Development of NTLA-1001: First-in-Class,
LNP-CRISPR/Cas9 Mediated Genome Editing Therapeutic for the
Treatment of ATTR.”
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: Amyloidosis Foundation and National Institutes of
Health)
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.
About Intellia Therapeutics
Intellia Therapeutics is a leading genome editing company
focused on developing 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, and through improved cell therapies that can treat cancer
and immunological diseases by replacing patients’ diseased cells.
The combination of deep scientific, technical and clinical
development experience, along with its leading intellectual
property portfolio, puts Intellia 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 and follow
us on Twitter @intelliatweets.
Forward-Looking Statements
This press release contains “forward-looking statements” of
Intellia Therapeutics, Inc. (“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, as well as our CRISPR/Cas9 intellectual property
portfolio; our ability to achieve stable or effective genome
editing; our ability to administer multiple doses of our
CRISPR/Cas9 product candidates; the potential timing and
advancement of our preclinical studies, including continuing
non-human primate studies for our Hereditary Transthyretin
Amyloidosis (“ATTR”) program and other programs, and clinical
trials; the timing and potential achievement of milestones to
advance our pipeline; our ability to replicate results achieved in
our preclinical studies in any future studies, including human
clinical trials; the potential development of other in vivo
or ex vivo cell therapeutics of all types, and those
targeting WT1 in particular, using CRISPR/Cas9 technology; our
ability to continue to conduct successful Investigational New Drug
(“IND”) enabling studies of a lead ATTR development candidate and
subsequently submitting an IND application by the end of 2019 that
will be accepted by the regulatory agencies; our intent to present
additional data for organs beyond the liver, additional
insertion/repair data, and preclinical data in support of our
first ex vivo programs on immuno-oncology and
autoimmune/inflammation indications during 2018; the expansion of
our fully automated bioinformatics platform; our ability to advance
a development candidate for a second indication by late 2018; our
potential ability to conduct a pre-IND meeting with the U.S.
Food and Drug Administration (“FDA”) for ATTR; the
intellectual property position and strategy of Intellia’s licensors
or other parties from which it derives rights; actions by
government agencies; the impact of our collaborations on our
development programs; the potential timing of regulatory filings
regarding our development programs; the potential commercialization
opportunities, including value and market, for product candidates;
our expectations regarding our uses of capital, expenses, future
accumulated deficit and other 2018 financial results; and our
ability to fund operations through mid-2020.
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.
Intellia Contacts:
Media:Jennifer Mound SmoterSenior Vice
PresidentExternal Affairs & Communications+1
857-706-1071jenn.smoter@intelliatx.com
Lynnea OlivarezAssociate DirectorExternal Affairs &
Communications+1 956-330-1917 lynnea.olivarez@intelliatx.com
Investors:Lindsey TrickettVice
PresidentInvestor Relations+1
857-285-6211lindsey.trickett@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