Intellia scientists advance CRISPR-mediated
targeted gene insertion in non-human primates
Intellia Therapeutics, Inc. (NASDAQ:NTLA), will present today new
data, including the first demonstration of targeted gene insertion
with CRISPR/Cas9 in the liver of non-human primates (NHPs), at the
22nd Annual Meeting of the American Society of Gene and Cell
Therapy (ASGCT), taking place April 29-May 2, 2019, in Washington,
D.C. Researchers also will present today new in vitro data from
Intellia’s lead engineered cell therapy program in acute myeloid
leukemia (AML). Later this week at the 2019 ASGCT meeting, Intellia
will present new data from its primary hyperoxaluria (PH1) program.
“The data we are presenting at ASGCT reflects our rapid progress
with Intellia’s modular CRISPR/Cas9 platform across a variety of in
vivo and engineered cell therapeutic applications,” said Intellia
President and Chief Executive Officer John Leonard, M.D. “Today’s
presentation of our most recent targeted gene insertion data
depicts Intellia’s second successful demonstration of
CRISPR-mediated gene editing in non-human primates, both in
collaboration with Regeneron Pharmaceuticals, Inc. The first was
through gene knockout in our transthyretin amyloidosis program and,
now, we have used our targeted insertion approach with Factor 9 as
a model gene. We also continue to make strides with our
collaborators at IRCCS Ospedale San Raffaele toward developing
engineered cell therapies for a variety of intractable cancers,
such as acute myeloid leukemia.”
Intellia’s First Demonstration of Targeted Gene
Insertion in NHPs
In a follow-up to Intellia’s presentation at the 2018 European
Society of Gene and Cell Therapy (ESGCT) meeting of the first
robust demonstration of CRISPR-mediated insertion of transgenes in
the liver of mice using Factor 9 (F9) as a model gene, the company
will present at ASGCT an advancement of its modular hybrid delivery
system, which combines Intellia’s lipid nanoparticle (LNP) platform
with an adeno-associated virus (AAV). F9 is a gene that
encodes FIX, a blood-clotting protein that is missing or defective
in hemophilia B patients.
In a collaboration between Intellia and Regeneron, researchers
delivered F9 DNA via a proprietary bi-directional
insertion template to demonstrate targeted gene
insertion in NHPs, resulting in circulating human
FIX protein levels within the normal range of human FIX
protein levels (3-5 μg/mL; source: Amiral et al., Clin. Chem.,
1984). Researchers observed circulating human FIX protein levels of
~3-4 μg/mL at day 14 after a single dose in an ongoing study, with
levels sustained through 28 days (~3-5 μg/mL).
Today, researchers additionally will share updated results
from an ongoing durability study, first reported in
October, that the circulating supratherapeutic human FIX
protein levels achieved in mice with Intellia’s hybrid LNP-AAV
delivery system have remained stable through 10 months of
observation. Further, researchers will show that they can regulate
FIX protein levels in mice by varying LNP doses, AAV doses or
insertion site.
Today’s presentation, titled “CRISPR/Cas9-Mediated Targeted
Insertion of Human F9 Achieves Therapeutic Circulating Protein
Levels in Mice and Non-Human Primates,” will be made by Hon-Ren
Huang, associate director, Vector Biology, Intellia. This
presentation will be accessible through the Events and
Presentations page of the Investor Relations section of Intellia’s
website at www.intelliatx.com.
Data Update from Intellia’s Acute Myeloid Leukemia
Program
Intellia and its research collaborator, IRCCS Ospedale San
Raffaele, will provide an update on the company’s lead engineered
cell therapy program in AML. Researchers will present new in vitro
data showing that CRISPR/Cas9 editing resulted in >98% knockout
of the endogenous T cell receptor (TCR), while achieving transfer
of various Wilms’ Tumor 1 (WT1)-specific TCRs into >95% of
isolated T cells. Researchers generated and tested a library of
TCRs with different epitope specificities and human leukocyte
antigen (HLA) restrictions, building on the data reported at the
2018 ESGCT meeting. Several lead TCRs restricted to the HLA-A*02:01
allele, a frequently expressed allele in the western hemisphere,
show the desired T cell characteristics, including high affinity,
epitope specificity and killing of a panel of primary leukemic
blast cells that expressed the WT1 antigen.
Intellia and OSR are collaborating to develop best-in-class
CRISPR-edited T cells directed to a specific epitope of WT1, a
tumor-associated antigen overexpressed across a wide range of
different tumor types and a known driver of leukocyte blasts in
hematological cancers. Intellia’s first cell therapy tumor target
is WT1 for the treatment of AML and other potential hematological
malignancies, as well as for solid tumors.
Today’s presentation, titled “Exploiting Clonal Tracking of
WT1-Specific T Cells to Generate a Library of Tumor-Specific T Cell
Receptors (TCR), for TCR Gene Editing of Acute Leukemia,” will be
made by Eliana Ruggiero, Ph.D., Experimental Hematology Unit,
Division of Immunology, Transplantation and Infectious Diseases,
IRCCS Ospedale San Raffaele, Italy.
Additional In Vivo Data to be Presented at the 2019
ASGCT Meeting
Intellia’s third oral presentation at the 2019 ASGCT Meeting
will take place later this week, on Thur., May 2, 2019. In a
presentation titled “CRISPR/Cas9-Mediated Gene Knockout to Address
Primary Hyperoxaluria,” the company will provide information
demonstrating successful independent knockout of two targets of
interest, lactate dehydrogenase A (LDHA) and hydroxyacid oxidase 1
(HAO1), to address PH1 in a PH1 mouse model.
The data shows the continued progression of Intellia’s modular
platform capability using CRISPR to knock out liver gene targets.
The data being presented includes results from an ongoing
collaboration with researchers at the University of Alabama at
Birmingham.
About Intellia Therapeutics
Intellia Therapeutics is a leading genome editing company
focused on developing curative therapeutics using the CRISPR/Cas9
system. Intellia believes the CRISPR/Cas9 technology has the
potential to transform medicine by permanently editing
disease-associated genes in the human body with a single treatment
course, and through improved cell therapies that can treat cancer
and immunological diseases, or can replace patients’ diseased
cells. The combination of deep scientific, technical and clinical
development experience, along with its leading intellectual
property portfolio, puts Intellia in a unique position to unlock
broad therapeutic applications of the CRISPR/Cas9 technology and
create a new class of therapeutic products. Learn more
about Intellia Therapeutics and CRISPR/Cas9
at intelliatx.com and follow us on Twitter
@intelliatweets.
Forward-Looking Statements
This press release contains "forward-looking statements" of
Intellia 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 our
ability to advance and expand the CRISPR/Cas9 technology to develop
human therapeutic products, as well as our CRISPR/Cas9 intellectual
property portfolio; our ability to achieve stable or effective
genome editing; our ability to perform genomic editing, such as
knock-out and insertion, to treat disease by modulating, replacing
or correcting genetic function; our ability to effectively
administer one dose or multiple doses of our CRISPR/Cas9-based
product candidates; the potential timing and advancement of our
preclinical studies, including continuing non-human primate studies
for our Transthyretin Amyloidosis (“ATTR”) program and other
studies for our other programs (such as, alpha-1 antitrypsin
deficiency (“AATD”), primary hyperoxaluria type 1 (“PH1”), and
acute myeloid leukemia (“AML”)), and clinical trials; our ability
to replicate results achieved in our preclinical studies in any
future studies, including human clinical trials; the potential
development of ex vivo cell therapeutics of all types using
CRISPR/Cas9 technology, including using Wilms’ Tumor 1 (“WT1”) to
develop cell therapies for the treatment of AML; our ability to
commence IND-enabling studies of a lead TTR development candidate
in 2019 and subsequently submitting an Investigational New Drug
application; our intent to present additional data for our liver
programs, organs beyond the liver, additional insertion/repair
data, and preclinical data in support of our in vivo programs,
including TTR and PH1, as well as our ex vivo programs on
immuno-oncology, including AML, during 2019; our ability to
nominate a development candidate for an ex vivo program, as well as
a second in vivo indication, in 2019; the intellectual property
position and strategy of Intellia, Intellia’s licensors and other
third parties from which Intellia derives rights; actions by
government agencies; the impact of our collaborations on our
development programs; the potential timing of regulatory filings
regarding our development programs; the potential commercialization
opportunities, including value and market, for our product
candidates; our expectations regarding our uses of capital,
expenses, future accumulated deficit and other 2019 financial
results; and our ability to fund operations into 2021.
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 our
intellectual property position; risks related to the ability of our
licensors to protect and maintain their intellectual property
position; uncertainties related to the initiation and conduct of
studies and other development requirements for our 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 will be predictive
of future results in connection with future studies; and the risk
that Intellia’s collaborations with Novartis, Regeneron, IRCCS
Ospedale San Raffaele or 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 filed
with the Securities and Exchange Commission, 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 Therapeutics undertakes no duty to
update this information unless required by law.
Intellia Contacts:
Media:Jennifer Mound SmoterSenior Vice
PresidentExternal Affairs & Communications+1
857-706-1071jenn.smoter@intelliatx.com
Lynnea OlivarezAssociate DirectorExternal Affairs &
Communications+1 956-330-1917lynnea.olivarez@intelliatx.com
Investors:Lina LiSenior ManagerInvestor
Relations+1 857-706-1612lina.li@intelliatx.com
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