Intellia Therapeutics Announces First Patient Dosed in Phase 1/2 Clinical Trial of NTLA-2002 for the Treatment of Hereditary Angioedema
13 Décembre 2021 - 1:30PM
Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage
genome editing company focused on developing curative therapeutics
using CRISPR/Cas9 technology both in vivo and ex
vivo, today announced that the first patient has been dosed with
NTLA-2002, the company’s in vivo CRISPR/Cas9 genome editing
candidate being developed as a single-dose therapy to prevent
attacks in people living with hereditary angioedema (HAE).
NTLA-2002 is a systemically administered therapy designed to
inactivate the target gene kallikrein B1 (KLKB1) to reduce plasma
kallikrein activity and thus prevent HAE attacks.
“HAE is a genetic disorder that can cause painful and
life-threatening inflammatory attacks, and currently available
chronic therapies have a high treatment burden,” said Intellia
President and Chief Executive Officer John Leonard, M.D. “With the
progress of our first-in-human clinical study evaluating NTLA-2002
for people living with HAE, we look forward to beginning clinical
testing as we aim to develop a single-dose treatment for these
patients.”
About the NTLA-2002 Clinical Program
Intellia’s multi-national Phase 1/2 study will evaluate 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 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 Therapeutics
Intellia Therapeutics, a leading clinical-stage genome editing
company, is developing novel, potentially curative therapeutics
using CRISPR/Cas9 technology. To fully realize the transformative
potential of CRISPR/Cas9, Intellia is pursuing two primary
approaches. The company’s in vivo programs use intravenously
administered CRISPR as the therapy, leveraging proprietary delivery
technology to enable 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 CRISPR/Cas9 to create new classes of genetic medicine. Learn
more at intelliatx.com. Follow us on Twitter @intelliatweets.
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: being able to complete clinical studies
for NTLA-2002 for the treatment of hereditary angioedema (“HAE”)
pursuant to its clinical trial applications (“CTA”), including
submitting additional regulatory applications in other countries;
advancement and expansion of its CRISPR/Cas9 technology to develop
human therapeutic products, as well as its ability to maintain and
expand its related intellectual property portfolio; ability to
demonstrate its platform’s modularity and replicate or apply
results achieved in preclinical studies, including those in its
transthyretin amyloidosis and HAE programs, in any future studies,
including human clinical trials; expectations of the potential
impact of the coronavirus disease 2019 pandemic on strategy, future
operations and timing of its clinical trials or IND submissions;
statements regarding the timing of regulatory filings regarding its
development programs; use of capital, expenses, future accumulated
deficit and other 2021 financial results or in the future; and
ability to fund operations beyond the next 24 months.
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 its
intellectual property position; risks related to Intellia’s
relationship with third parties, including its licensors and
licensees; risks related to the ability of its licensors to protect
and maintain their intellectual property position; uncertainties
related to the authorization, initiation and conduct of studies and
other development requirements for its 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 or clinical studies will not be
predictive of future results in connection with future studies; and
the risk that Intellia’s collaborations with Regeneron or its other
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 quarterly report on 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. 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 LiDirector, Investor
Relations+1-857-706-1612lina.li@intelliatx.com
Media:
Matt CrensonTen Bridge
Communications+1-917-640-7930media@intelliatx.commcrenson@tenbridgecommunications.com
Intellia Therapeutics (NASDAQ:NTLA)
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