Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading genome editing
company focused on developing curative therapeutics using
CRISPR/Cas9 technology both in vivo and ex
vivo, announced that five abstracts were accepted for the 23rd
Annual Meeting of the American Society of Gene and Cell Therapy
(ASGCT), taking place virtually from May 12-15, 2020. Abstracts are
available on the ASGCT website.
Intellia’s data include important updates about the company’s
progress and platform development activities:
Oral Presentations
“Enhanced tgTCR T Cell Product Attributes Through
Process Improvement of CRISPR/Cas9 Engineering” As a
follow-on to data presented at Keystone Symposia’s Engineering the
Genome Conference this past February, Intellia’s ASGCT presentation
will focus on process improvements in its CRISPR/Cas9-based
engineering to deliver T cell therapies with high levels of
editing, achieving robust levels of expansion, desirable memory
phenotypes, improved function and reduced translocations. These
platform advances support NTLA-5001, which is Intellia’s first
wholly owned engineered T cell therapy development candidate.
NTLA-5001 utilizes a T cell receptor (TCR)-directed approach to
target the Wilms’ Tumor 1 (WT1) antigen, obtained through the
ongoing research collaboration with IRCCS Ospedale San Raffaele,
which is over-expressed in many hematologic and solid tumors.
Intellia will focus its initial clinical investigation of NLTA-5001
on acute myeloid leukemia (AML), while exploring in ongoing
preclinical studies its application in solid tumors. Intellia plans
to submit an IND for NTLA-5001 for the treatment of AML in the
first half of 2021.
Presenter: Aaron Prodeus, Ph.D., senior
scientist, Cell Therapy Abstract number: 35
Session: CAR T-Cell Therapies I
Presentation date/time: Tuesday, May 12, 2020,
from 11:45 a.m.-12 p.m. ET
“Exploiting CRISPR-Genome Editing and WT1-Specific T
Cell Receptors to Redirect T Lymphocytes Against Acute Myeloid
Leukemia” This presentation will focus on Intellia’s
ongoing research collaboration with IRCCS Ospedale San Raffaele to
apply CRISPR/Cas9 technology to develop engineered T cell therapies
to address intractable cancers, such as AML. Researchers will also
present data showing a significant decrease in AML tumor burden in
mice treated with the WT1-specific TCRs, which are incorporated
into Intellia’s lead NTLA-5001 candidate for the treatment of
AML.
Presenter: Eliana Ruggiero, Ph.D., Experimental
Hematology Unit, Division of Immunology, Transplantation and
Infectious Diseases, IRCCS Ospedale San Raffaele, Italy
Abstract number: 78 Session: CAR
T and Other Engineered T Cells Targeting Hematological Malignancies
Presentation date/time: Tuesday, May 12, 2020,
from 3:45-4 p.m. ET
“CRISPR/Cas9-Mediated Gene Knockout of KLKB1 to Treat
Hereditary Angioedema” Intellia will present data updates
on its potential hereditary angioedema (HAE) therapy, which uses
the company’s modular lipid nanoparticle (LNP)-based CRISPR/Cas9
delivery system to knock out the prekallikrein B1 (KLKB1) gene and
reduce kallikrein activity. HAE is a rare genetic disorder
characterized by recurring and unpredictable severe swelling
attacks in various parts of the body, and is significantly
debilitating or even fatal in certain cases. This presentation will
include results from the company’s ongoing collaboration with
researchers at Regeneron and also will build on initial data
released at Keystone Symposia’s Engineering the Genome Conference
in February. Intellia expects to nominate a development candidate
for HAE in the first half of 2020.
Presenter: Jessica Seitzer, director, Genomics
Abstract number: 1320 Session:
Gene Regulation and Delivery Technologies Presentation
date/time: Friday, May 15, 2020, from 10:30-10:45 a.m.
ET
Poster Presentations
“Validation of CRISPR/Cas9 Off-Target Discovery Profiles
from In Silico Prediction, Cell-Based and Biochemical-Based Assays
with Targeted Off-Target Sequencing” This presentation
will highlight Intellia’s approach to assess off-target activity to
identify highly specific CRISPR/Cas9 guides. Researchers
demonstrated that potential off-target editing profiles discovered
through empirical data from biochemical approaches were the most
sensitive and accurate.
Presenter: Nishit Patel, Ph.D., senior
associate scientist, Informatics, Computational Biology and
Genomics Abstract number: 203 Session
date/time: Tuesday, May 12, 2020, from 5:30-6:30 p.m.
ET
“In Vivo Model Development for Genome-Edited T Cell
Therapeutics”
Researchers will present improved murine models used to assess
safety, efficacy and persistence of therapeutic modalities in
cell-based therapies. Together with biotechnology company Taconic
Biosciences, Intellia developed two in vivo mouse models, which
allow for enhanced monitoring of graft-versus-host disease and
human natural killer cytotoxicity.
Presenter: Yong Zhang, Ph.D., principal
scientist, Cell Therapy Abstract number: 1174
Session date/time: Thursday, May 14, 2020, from
5:30-6:30 p.m. ET
Workshop and Symposia
“Building a Modular CRISPR/Cas9 Platform for Human
Therapeutic Applications” Intellia will participate in the
ASGCT Translational Science Genome Editing Pre-Meeting Workshop,
which will include an overview on important factors to consider in
building a modular CRISPR/Cas9 platform for human therapeutic
applications.
Presenter: Jessica Seitzer, director, Genomics
Session: Gene Editing Workshop
Presentation date/time: Monday, May 11, 2020, from
4:10-4:30 p.m. ET
Industry Sponsored Symposia
Intellia will participate in an industry lunchtime panel
discussion during ASGCT.
Panelist: Laura Sepp-Lorenzino, Ph.D., chief
scientific officer Panel date/time: Wednesday, May
13, 2020, from 12-1:30 p.m. ET
About Intellia Therapeutics
Intellia Therapeutics is a leading genome editing company
focused on developing proprietary, 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 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 ability to
advance and expand the CRISPR/Cas9 technology to develop human
therapeutic products that meet the regulatory requirements in the
relevant jurisdictions, as well as our CRISPR/Cas9 intellectual
property portfolio; our ability to achieve stable or effective
genome editing; our ability to effectively administer one dose or
multiple doses of our CRISPR/Cas9 product candidates; the potential
timing and advancement of our preclinical studies, including
non-human primate studies for our transthyretin amyloidosis
(“ATTR”) program (“NTLA-2001”) and our hereditary angioedema
(“HAE”) program, and other studies for these and other programs,
including preclinical and human clinical trials; the ability to
demonstrate our platform’s modularity and replicate or apply
results achieved in our preclinical studies, including those in our
ATTR, HAE and Wilms’ Tumor 1 (“WT1")/acute myeloid leukemia (“AML”)
programs or research projects, in any future studies, including
human clinical trials; our ability to generate data and replicate
results relating to enhancements to our proprietary lipid
nanoparticle (“LNP”) technology, including its formulation and
components, in preclinical or clinical studies, or that any
enhancements will result in an improved product candidate profile;
the potential development of other in vivo or ex vivo cell
therapeutics of all types, and those targeting WT1 in particular,
using CRISPR/Cas9 technology; our plans to submit an IND
application for NTLA-2001 for ATTR in the second half of 2020, as
well as for NTLA-5001, our first T cell receptor (“TCR”)-directed
engineered cell therapy development candidate for our AML program
in the first half of 2021; our plans to nominate a development
candidate for our HAE program in the first half of 2020; our
expectations regarding potential patient populations that may be
addressed by each of our programs; actions by government agencies;
our growth as a company and the anticipated contribution of the
members of our board of directors and our executives to our
operations and progress; the impact of our collaborations on our
research and development programs; the potential timing of
regulatory filings regarding our development programs; the
potential commercial opportunities, including value and market, for
our product candidates; our expectations regarding our use of
capital and other financial results during 2020; and our ability to
fund operations through the end of 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 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 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, OSR 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 Olivarez
Director
External Affairs & Communications
+1 956-330-1917
lynnea.olivarez@intelliatx.com
Investors:
Lina Li
Associate Director
Investor Relations
+1 857-706-1612
lina.li@intelliatx.com
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