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 new data supporting novel capabilities of its
CRISPR/Cas9 genome editing platform, which the Company plans to
leverage for the development of future therapeutic candidates. The
data shared showed that Intellia’s allogeneic platform, leveraging
a novel combination of sequential gene edits, can prevent immune
rejection of allogeneic T cells in in vitro and in vivo models for
future application in TCR-T and CAR-T therapy. Additionally, data
highlighted that lipid nanoparticles (LNPs) can replace
electroporation for delivery of CRISPR/Cas9 gene edits to T cells,
avoiding the risk of chromosomal translocations observed when
multiple edits are performed simultaneously, as well as the
negative effect of electroporation on T cell health. Finally,
results from an ongoing study demonstrated proof-of-concept in
non-human primates (NHPs) for in vivo gene insertion and knockout
for the treatment of alpha-1 antitrypsin deficiency (AATD), which
resulted in sustained production of normal human levels of healthy
alpha-1 antitrypsin (A1AT) protein and reduction of the endogenous
disease-associated protein. The data were presented at the 29th
Annual Congress of the European Society of Gene & Cell Therapy
(ESGCT) meeting, taking place virtually from October 19 – 22, 2021.
“Preclinical data presented at ESGCT’s Annual Congress show that
using our proprietary genome editing platform, Intellia is able to
accomplish multiple CRISPR/Cas9 edits in both in vivo and ex vivo
applications, advancing our efforts to develop treatments for
challenging genetic diseases like alpha-1 antitrypsin deficiency
and to potentially expand both the effectiveness and availability
of engineered cell therapies for the treatment of cancer and
autoimmune diseases,” said Intellia President and Chief Executive
Officer John Leonard, M.D. “This important preclinical work
supports our mission as we look ahead to initiating a
first-in-human study of NTLA-5001, our first ex vivo candidate, in
patients with acute myeloid leukemia, and nominating at least one
new development candidate before the end of this year as well as
additional candidates in 2022.”
Proprietary allogeneic solution that can be readily
deployed for TCR-T and CAR-T therapy
Key immunological challenges remain unaddressed by allogeneic,
or “off-the-shelf”, T cell therapies currently in development for
cancer treatment. Leveraging Intellia’s CRISPR/Cas9 platform and an
innovative sequential gene editing process, the Company has
developed a proprietary allogeneic solution that may avoid the need
for long-term or aggressive immunosuppressive regimens and could be
readily deployed for TCR-T and CAR-T therapy. The data shared at
ESGCT demonstrate that a novel combination of targeted gene edits
protected therapeutic T cells from host T cell as well as NK
cell-mediated killing in in vitro and in vivo mouse models.
Furthermore, these engineered cells showed no impairment in their
tumor-killing ability in in vitro assays compared to their
autologous counterparts. As part of these efforts, Intellia intends
to nominate its first allogeneic cell therapy development candidate
by the first half of 2022.
Using lipid nanoparticles to engineer next-generation
CRISPR-based cell therapies
Adoptive cell therapies have been successful in certain cancers
but have encountered technical and biological barriers, such as
reliance on electroporation for editing of T cells, which impacts T
cell viability, expansion and gene expression, and can lead to
chromosomal translocations when used to introduce multiple
simultaneous gene edits. At ESGCT, Intellia presented data
demonstrating the use of LNPs to engineer CRISPR-based T cell
therapies without the need for electroporation, advancing a robust,
modular and scalable platform with the potential to enable future
allogeneic and solid tumor therapies requiring multiple genome
edits. The data showed that T cells engineered with LNPs showed
efficient editing rates, with improved cell properties and
performance both in vitro and in vivo, as compared to
electroporation. In addition, the lower toxicity associated with
LNP delivery allows Intellia’s platform to produce sequentially
edited T cells with high efficiency, faster expansion and minimal
translocations as compared to electroporation – demonstrated by
targeting up to five or more loci (four knockouts and one to two
targeted, in-locus insertions). The data support the ability of
this platform to be used for a variety of targeting modalities,
including CARs and TCRs, to support both autologous or allogeneic T
cell candidates, including those requiring multiple edits to
address immune rejection and activity in solid or other
immune-suppressive tumors. This LNP-based approach is already being
used for NTLA-5001, the Company’s first wholly owned ex vivo genome
editing candidate, which is in development for acute myeloid
leukemia. Intellia expects to initiate patient screening for the
Phase 1/2a study of NTLA-5001 by year-end.
Tailored genome editing approach offers potential to
independently treat liver and lung manifestations of alpha-1
antitrypsin deficiency (AATD)
New data shared at ESGCT represent the first reported
demonstration of consecutive in vivo gene insertion and gene
knockout in NHPs. This is an important step toward treating
diseases such as AATD, which can manifest as lung disease (due to
insufficient functional A1AT protein levels) or liver disease (due
to accumulation of mutant A1AT protein) and thus require either
inserting a functional gene, removing a disease-associated gene or
both. The Company reported data showing that insertion of a healthy
form of the SERPINA1 gene, which encodes the A1AT protein, led to
normal human A1AT levels in NHPs which were durable through 52
weeks in an ongoing study. Intellia has also now tested the ability
to knock out the endogenous cynomolgus SERPINA1 gene while leaving
the inserted healthy human version intact. This insertion followed
by knockout led to the continued production of normal human levels
of functioning A1AT protein -- substantially higher than what has
been seen with other treatment approaches -- as well as reduction
of the disease-associated protein. Together, these data support the
ability of Intellia’s in vivo genome editing platform to address
the lung and/or liver manifestations of AATD as needed for a given
patient.
Presentations will be available on Intellia’s website at
www.intelliatx.com.
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, 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 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: ability to initiate a clinical trial
for NTLA-5001 for the treatment of acute myeloid leukemia (“AML”)
by the end of 2021; ability to generate data to demonstrate
NTLA-5001 as a potential best-in-class engineered T cell therapy
designed to treat all genetic subtypes of AML; plans to evaluate in
preclinical studies the potential use of NTLA-5001 to treat Wilms’
Tumor 1 (“WT1”)-positive solid tumors; plans to advance and
complete preclinical studies for our research programs; development
of our modular platform to advance our complex genome editing
capabilities; further development of our proprietary genome editing
tools for research and therapeutic development, including
sequential editing; presentation of additional data at upcoming
scientific conferences, and other preclinical data in 2021;
advancement and expansion of our CRISPR/Cas9 technology to develop
human therapeutic products; ability to select an allogeneic cell
therapy development candidate in the first half of 2022; ability to
maintain and expand our related intellectual property portfolio,
and avoid or acquire rights to valid intellectual property of third
parties; ability to demonstrate our platform’s modularity and
replicate or apply results achieved in preclinical studies,
including those in our AML program, in any future studies,
including human clinical trials; ability to develop other in vivo
or ex vivo cell therapeutics of all types, and those targeting WT1
in AML in particular, 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 our
collaborations on our development programs, statements regarding
the timing of regulatory filings and clinical trial execution,
including dosing of patients, regarding our development programs;
potential commercial opportunities, including value and market, for
our product candidates; our expectations regarding our use of
capital and other financial results during 2021; and our 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:Lisa QuTen Bridge
Communications+1-678-662-9166media@intelliatx.comlqu@tenbridgecommunications.com
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