Intellia Therapeutics, Inc. (NASDAQ:NTLA) is presenting new
data demonstrating the persistence of in vivo CRISPR/Cas9 edits to
either reduce a disease-causing protein or restore a functional
protein, in a mouse model of accelerated liver regeneration. These
data will be included in the company’s invited talk at this year’s
16th Annual Meeting of the Oligonucleotide Therapeutics Society
(OTS), taking place virtually from September 27-30, 2020.
“As we prepare to enter the clinic with NTLA-2001, our first
systemic treatment, we are extremely encouraged by the durability
Intellia scientists observed with both gene knockout as well as
targeted insertion in a partial hepatectomy (PHx) mouse model. The
persistence of these edits and durable effects further support our
technology’s ability to develop potentially curative single-course
therapies, and provide clear differentiation from chronic
treatments and traditional AAV gene therapy,” said Intellia Chief
Scientific Officer Laura Sepp-Lorenzino, Ph.D. “Our modular
delivery platform is enabling us to rapidly advance multiple
product candidates in parallel – and to ensure that the therapeutic
impact will be long-lasting for patients in need.”
The OTS talk titled, “A Modular CRISPR/Cas9 Genome Editing
Platform for Durable Therapeutic Knockout and Targeted Gene
Insertion Applications,” will be given today at 10 a.m. ET by
Anthony Forget, Ph.D., senior director of genome editing at
Intellia. Click here to view the presentation slides.
Persistent In Vivo
Liver Gene Knockouts and Corresponding Protein
Reduction Achieved Employing
Intellia’s Modular
Platform
Accelerated hepatocyte turnover following PHx in mice was
employed to assess the durability of gene knockout and insertion
edits. After resection of 2/3 of the liver, and subsequent
full-liver regeneration, genome edits and corresponding protein
levels were unchanged, supporting the permanent nature of the edit,
which is carried through when liver cells proliferate. This update
builds upon previously reported data of the edits’ year-long
durability demonstrated in rodents and non-human primates for
Intellia’s liver knockout therapeutic candidates, transthyretin
amyloidosis (ATTR) and hereditary angioedema (HAE).
Intellia’s lead in vivo candidate, NTLA-2001, is being studied
as a single-course treatment for ATTR using the company’s liver
knockout editing approach. NTLA-2001 employs a proprietary lipid
nanoparticle (LNP) delivery system and is designed to permanently
inactivate the disease-causing gene in the liver. Knocking out the
transthyretin (TTR) gene may lead to a lifelong reduction of TTR
protein and associated ATTR symptoms. The company expects to dose
the first ATTR patient by year-end with NTLA-2001, which is part of
a co-development/co-promotion agreement between Intellia, the lead
development and commercialization party, and Regeneron
Pharmaceuticals, Inc.
The modularity of Intellia’s proprietary platform allows
Intellia to modify only a single component, the guide RNA sequence,
to develop other in vivo therapies for additional targets of
interest. Intellia also is applying its LNP delivery system to
develop NTLA-2002 to treat HAE by targeting and knocking out the
prekallikrein B1 (KLKB1) gene in the liver. The company anticipates
submitting an Investigational New Drug (IND) application or
IND-equivalent for NTLA-2002 in the second half of 2021.
CRISPR-Mediated Targeted Gene Insertion
Has Demonstrated
Advantages Over AAV-Based Gene Therapy
Approaches
Intellia will present additional data highlighting the potential
of its liver insertion platform, as exemplified by the company’s
hemophilia B program. Intellia and Regeneron, the lead party, are
co-developing potential hemophilia A and B CRISPR/Cas9-based
treatments using their jointly developed targeted transgene
insertion capabilities.
Data shows the company achieved circulating activity levels for
Factor IX (FIX), a blood-clotting protein that is missing or
defective in hemophilia B patients, ranging from normal levels
(50-150%, Source: National Hemophilia Foundation) to
supratherapeutic levels in a six-week non-human primate study.
Insertion efficiency and transgene expression can be controlled by
three independent factors: the precise insertion site targeted by
the guide RNA; the dose of LNP, which delivers the CRISPR
machinery; and the amount of the promoter-less DNA template donor
that encodes the transgene’s DNA sequence for insertion. These
levers allow for optimization of transgene expression according to
the desired therapeutic target levels and may allow for higher
levels of expression than those attained by traditional
adeno-associated virus (AAV) gene therapy, if required.
Furthermore, unlike traditional gene therapy, for which a
significant loss (over 80%) in transgene expression was observed
after in vivo PHx and liver regeneration, Intellia’s targeted gene
insertion approach yielded durable edits as cells proliferate, with
no significant loss in expression. These findings support the
development of gene insertion therapies.
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
producing single-course therapeutics that permanently edit and
correct disease-associated genes, and creating enhanced engineered
cells that can treat oncological and immunological diseases.
Intellia’s combination of deep scientific, technical and clinical
development experience, along with its leading intellectual
property portfolio, puts it in a unique position to unlock broad
therapeutic applications of the CRISPR/Cas9 technology and create
new classes 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 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: receiving authorization to initiate
clinical studies for NTLA-2001 for the treatment of transthyretin
amyloidosis (“ATTR”) pursuant to its clinical trial application
(“CTA”) or similar regulatory applications, and the planned dosing
of first patients by the end of 2020; plans to complete
manufacturing activities and submit an IND or equivalent regulatory
filing for NTLA-2002, a development candidate for its hereditary
angioedema (“HAE”) program in the second half of 2021; plans to
advance and complete preclinical studies, including any necessary
non-human primate studies, for its ATTR program, HAE program,
hemophilia A, hemophilia B, and other in vivo and ex vivo programs;
development of a proprietary LNP/AAV hybrid delivery system, as
well as its modular platform to advance its complex genome editing
capabilities, such as gene insertion, as well as knockout editing
capabilities; presentation of additional data at upcoming
scientific conferences, and other preclinical data in 2020;
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
ATTR, HAE, hemophilia A and hemophilia B programs, in any future
studies, including human clinical trials; ability to develop other
in vivo or ex vivo cell therapeutics of all types using CRISPR/Cas9
technology; expectations of the potential impact of the coronavirus
disease 2019 pandemic on strategy, future operations and timing of
its clinical trials or IND submissions; ability to optimize the
impact of its collaborations on its development programs, including
but not limited to its collaborations with Regeneron, including its
co-development programs for ATTR, hemophilia A and hemophilia B;
statements regarding the timing of regulatory filings regarding its
development programs; use of capital, expenses, future accumulated
deficit and other 2020 financial results or in the future; and
ability to fund operations at least through 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 annual report on Form 10-K 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:
Media:Lynnea OlivarezDirectorExternal Affairs
& Communications+1 956-330-1917
lynnea.olivarez@intelliatx.com
Investors:Lina LiAssociate DirectorInvestor
Relations+1 857-706-1612lina.li@intelliatx.com
Intellia Therapeutics (NASDAQ:NTLA)
Graphique Historique de l'Action
De Août 2024 à Sept 2024
Intellia Therapeutics (NASDAQ:NTLA)
Graphique Historique de l'Action
De Sept 2023 à Sept 2024