Intellia Therapeutics Awarded Innovation Passport in the United Kingdom for NTLA-2002, an Investigational Genome Editing Treatment for Hereditary Angioedema
11 Janvier 2023 - 1:30PM
Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage
genome editing company focused on developing potentially curative
therapies leveraging CRISPR-based technologies, today announced the
U.K. Medicines and Healthcare products Regulatory Agency (MHRA) has
awarded the Innovation Passport for NTLA-2002, an in vivo genome
editing candidate being developed for the treatment of hereditary
angioedema (HAE). The Innovation Passport is the point of entry
into the U.K.’s Innovative Licensing and Access Pathway (ILAP),
which is designed to accelerate time to market and facilitate
patient access to innovative medicines.
“With the high treatment burden of currently available chronic
therapies for hereditary angioedema, we are pleased to receive the
ILAP designation, which will enable Intellia to further accelerate
the clinical development of NTLA-2002,” said Intellia President and
Chief Executive Officer John Leonard, M.D. “We expect to begin the
Phase 2 portion of the NTLA-2002 clinical study in the first half
of this year, and we look forward to working with the U.K. and
other regulatory agencies to bring this investigational single-dose
genome editing treatment to patients as quickly as possible.”
The Phase 1/2 study evaluating NTLA-2002 in adults with Type I
or Type II HAE is currently ongoing, with the Phase 2 portion
expected to initiate in the first half of 2023.
Delivered in partnership by the All Wales Therapeutics and
Toxicology Centre (AWTTC), the Medicines and Healthcare products
Regulatory Agency (MHRA), the National Institute for Health and
Care Excellence (NICE) and the Scottish Medicines Consortium (SMC),
the Innovation Passport prioritizes innovative medicines in early
clinical development stages for the treatment of diseases with a
significant patient or public health need. Benefits of the ILAP
include access to a Target Development Profile, which defines key
regulatory and development features and creates a roadmap for
advancement through regulatory approval.
About the NTLA-2002 Clinical Program
Intellia’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, accelerate, and
complete clinical studies for NTLA-2002 for the treatment of
hereditary angioedema (HAE); its ability to begin the Phase 2
portion of the NTLA-2002 Phase 1/2 clinical study in the first half
of 2023; its ability to generate data to demonstrate NTLA-2002 as a
potential single-dose treatment for HAE; 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 replicate or apply results achieved in preclinical
and clinical 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 NTLA-2002; the risk that NTLA-2002
will not be successfully developed and commercialized; and 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. 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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