Intellia Therapeutics to Present Interim Clinical Data from Ongoing Phase 1/2 Study of NTLA-2002 for the Treatment of Hereditary Angioedema at the 2022 Bradykinin Symposium
23 Août 2022 - 3:59PM
Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage
genome editing company focused on developing potentially curative
therapeutics leveraging CRISPR-based technologies, today announced
that an abstract featuring interim clinical data from the Phase 1/2
study of NTLA-2002 has been selected for an oral presentation at
the 2022 Bradykinin Symposium, taking place September 15-16 in
Berlin, Germany. NTLA-2002 is an investigational in vivo
CRISPR/Cas9 therapy in development as a single-dose treatment to
prevent angioedema attacks in people living with hereditary
angioedema (HAE). The presentation will include interim safety,
kallikrein reduction and attack rate data from the ongoing
dose-escalation portion of Intellia’s first-in-human study of
NTLA-2002.
Presentation DetailsTitle: "In
vivo CRISPR/Cas9 editing of KLKB1 in Patients with HAE”
Date and Time: Friday, September 16, 2022, from
10:55 – 11:05 a.m. CESTSession: Session V – Short
Oral PresentationsPresenter: Dr. Hilary Longhurst,
M.D., Ph.D., FRCP, FRCPath, Senior Medical Officer at Auckland
District Health Board and Honorary Associate Professor at
University of Auckland, New Zealand, the trial’s principal
investigator in New Zealand
Intellia Therapeutics Investor EventIntellia
plans to host a webcast to review the presented clinical data at
the 2022 Bradykinin Symposium. The Company will announce details
for the event in September.
About the NTLA-2002 Clinical ProgramIntellia’s
multi-national Phase 1/2 study is evaluating the safety,
tolerability, pharmacokinetics and pharmacodynamics of NTLA-2002 in
adults with Type I or Type II hereditary angioedema (HAE). This
includes the measurement of plasma kallikrein protein levels and
activity as determined by HAE attack rate measures. The Phase 1
portion of the study is an open-label, single-ascending dose design
used to identify up to two dose levels of NTLA-2002 that will be
further evaluated in the randomized, placebo-controlled Phase 2
portion of the study. This Phase 1/2 study will identify the dose
of NTLA-2002 for use in future studies. Visit
clinicaltrials.gov (NCT05120830) for more details.
About NTLA-2002
Based on Nobel Prize-winning CRISPR/Cas9 technology, NTLA-2002
is the first single-dose investigational treatment being explored
in clinical trials for the potential to continuously reduce
kallikrein activity and prevent attacks in people living with
hereditary angioedema (HAE). NTLA-2002 is a wholly owned
investigational CRISPR therapeutic candidate designed to inactivate
the kallikrein B1 (KLKB1) gene, which encodes for
prekallikrein, the kallikrein precursor protein. NTLA-2002 is
Intellia’s second investigational CRISPR therapeutic candidate
to be administered systemically, by intravenous infusion, to edit
disease-causing genes inside the human body with a single dose of
treatment. Intellia’s proprietary non-viral platform deploys lipid
nanoparticles to deliver to the liver a two-part genome
editing system: guide RNA specific to the disease-causing
gene and messenger RNA that encodes the Cas9 enzyme, which
together carry out the precision editing.
About Hereditary Angioedema
Hereditary angioedema (HAE) is a rare, genetic disorder
characterized by severe, recurring and unpredictable inflammatory
attacks in various organs and tissues of the body, which can be
painful, debilitating and life-threatening. It is estimated that
one in 50,000 people are affected by HAE, and current treatment
options often include life-long therapies, which may require
chronic intravenous (IV) or subcutaneous (SC) administration as
often as twice per week, or daily oral administration to ensure
constant pathway suppression for disease control. Despite chronic
administration, breakthrough attacks still occur. Kallikrein
inhibition is a clinically validated strategy for the preventive
treatment of HAE attacks.
About Intellia TherapeuticsIntellia
Therapeutics, a leading clinical-stage genome editing company, is
developing novel, potentially curative therapeutics leveraging
CRISPR-based technologies. To fully realize the transformative
potential of CRISPR-based technologies, Intellia is pursuing two
primary approaches. The company’s in vivo programs use
intravenously administered CRISPR as the therapy, in which
proprietary delivery technology enables highly precise editing of
disease-causing genes directly within specific target tissues.
Intellia’s ex vivo programs use CRISPR to create the therapy by
using engineered human cells to treat cancer and autoimmune
diseases. Intellia’s deep scientific, technical and clinical
development experience, along with its robust intellectual property
portfolio, have enabled the company to take a leadership role in
harnessing the full potential of genome editing to create new
classes of genetic medicine. Learn more at intelliatx.com. Follow
us on Twitter @intelliatx.
Forward-Looking Statements
This press release contains “forward-looking statements” of
Intellia Therapeutics, Inc. (“Intellia” or the “Company”) 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 beliefs and
expectations regarding: its ability to conduct and complete
clinical studies for NTLA-2002 for the treatment of hereditary
angioedema (HAE); its ability to generate data to demonstrate
NTLA-2002 as a potential single-dose treatment for HAE, including
safety, kallikrein reduction and attack rate data; its ability to
develop its modular platform and full-spectrum approach to advance
its complex genome editing capabilities, including to apply its
proprietary CRISPR/Cas9 technology platform to additional product
candidates; the advancement and expansion of its CRISPR/Cas9
technology to develop human therapeutic products; its ability to
maintain and expand its related intellectual property portfolio,
and avoid or acquire rights to valid intellectual property of third
parties; its ability to demonstrate its platform’s modularity and
replicate or apply results achieved in preclinical studies,
including those in its NTLA-2002 program, in any future studies,
including human clinical trials; its ability to develop other in
vivo or ex vivo cell therapeutics of all types, and NTLA-2002 in
particular, using CRISPR/Cas9 technology; and the timing of
regulatory filings and clinical trial execution, including dosing
of patients.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 the successful enrollment
of patients in the Phase 1/2 study for NTLA-2002 for the treatment
of HAE; risks related to Intellia’s ability to protect and maintain
its intellectual property position; risks related to the
authorization, initiation and conduct of studies and other
development requirements, including manufacturing, for its in vivo
and ex vivo product candidates, including NTLA-2002; the risk that
any one or more of Intellia’s product candidates, including
NTLA-2002, will not be successfully developed and commercialized;
the risk that the results of preclinical studies or clinical
studies, including for NTLA-2002, will not be predictive of future
results in connection with future studies; and the risk that
Intellia’s will not be able to demonstrate its platform’s
modularity and replicate or apply results achieved in preclinical
studies to develop additional product candidates, including to
apply its proprietary CRISPR/Cas9 technology platform successfully
to additional product candidates. 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 report of Form 10-Q, as well as discussions
of potential risks, uncertainties and other important factors in
Intellia’s other filings with the Securities and Exchange
Commission (SEC). All information in this press release is as of
the date of the release, and Intellia undertakes no duty to update
this information unless required by law.
Intellia Contacts:
Investors:Ian KarpSenior Vice President,
Investor Relations and Corporate
Communications+1-857-449-4175ian.karp@intelliatx.com
Lina LiSenior Director, Investor Relations and Corporate
Communications +1-857-706-1612lina.li@intelliatx.com
Media:Rebecca SpaldingTen Bridge
Communications+1-646-509-3831media@intelliatx.com
rebecca@tenbridgecommunications.com
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