Intellia Therapeutics Presents New Preclinical Data Supporting Its CRISPR/Cas9-Engineered TCR-T Cell Treatment for Acute Myel...
05 Décembre 2020 - 4:00PM
Intellia Therapeutics, Inc. (NASDAQ:NTLA), is presenting new
preclinical data in support of NTLA-5001, the company’s wholly
owned Wilms’ Tumor 1 (WT1)-directed T cell receptor (TCR)-T cell
therapy candidate for the treatment of acute myeloid leukemia
(AML), at the 62nd American Society of Hematology (ASH) Annual
Meeting, taking place virtually from December 5-8, 2020. NTLA-5001
capitalizes on how natural T cells recognize and respond to tumors.
The target, WT1, is highly overexpressed in AML, a cancer of the
blood and bone marrow that is often fatal despite existing
treatments (NIH SEER Cancer Stat Facts: Leukemia – AML). The
new preclinical data being presented today highlight the faster
expansion and superior function of T cells manufactured by
Intellia’s proprietary approach, compared to a standard genome
editing process. Specifically, NTLA-5001’s lead TCR-T cells
resulted in significantly higher anti-tumor activity in mouse
models of acute leukemias than that observed in mice treated with
cells engineered using the standard process.
“NTLA-5001 is the first potential CRISPR-based cancer treatment
engineered using Intellia’s proprietary process. Based on our
preclinical results, we believe our process will result in a
pipeline of safer and more efficacious oncological products, with
reduced manufacturing time and, importantly, reduced vein-to-vein
time, compared to currently available approaches. Showing in vivo
efficacy in acute leukemia mouse models, as presented today at ASH,
is extremely encouraging and an important steppingstone to entering
the clinic next year,” said Intellia President and Chief Executive
Officer John Leonard, M.D. “In our first-in-human trial, we plan to
establish the safety and activity that will enable us to move
quickly to a pivotal investigation of NTLA-5001 for the treatment
of AML, which is the most common type of acute leukemia in
adults.”
NTLA-5001 is being developed using Intellia’s proprietary
process to treat AML patients regardless of the genetic subtype of
a patient’s leukemia. Intellia plans to submit an Investigational
New Drug (IND) application or equivalent for NTLA-5001 in the first
half of 2021, subject to the impact of the COVID-19 pandemic, with
the first-in-human trial planned to evaluate safety and activity in
patients with persistent or recurrent AML who have previously
received first-line therapies. Additional efforts are underway to
evaluate the potential use of NTLA-5001 to treat WT1-positive solid
tumors.
Presentation Details
Title: “NTLA-5001, a T Cell Product Candidate
with CRISPR-Based Targeted Insertion of a High-Avidity, Natural,
WT1-Specific TCR, Shows Efficacy in In Vivo Models of AML and
ALL”Publication Number: 1435 Session
Name: 703. Adoptive Immunotherapy: Poster I
Presenting Author: Birgit Schultes, Ph.D., vice
president of Intellia’s Cell Therapy group
With Intellia’s proprietary T cell engineering process,
CRISPR/Cas9 in combination with adeno-associated virus (AAV) is
used to insert a WT1-directed TCR in locus, while eliminating the
expression of the endogenous TCRs. Benefits of Intellia’s approach
include the following:
- Intellia’s proprietary T cell engineering process enables
multiple, sequential gene edits and is a significant improvement
over standard engineering processes commonly used to introduce
proteins and nucleic acids into cells.
- Sequential editing maintains high T cell viability and may
result in safer T cell products because treated cells have minimal
levels of translocations, similar to unedited cells, and do not
cause graft-versus-host disease (GvHD).
- The observed faster T cell expansion
with favorable T cell memory phenotype could lead to a reduced
vein-to-vein time and better T cell persistence in patients,
respectively.
T cells engineered using Intellia’s proprietary process to
express the lead TCR to the WT137-45 epitope are efficacious,
durable and safe in vivo in gold-standard mouse models of AML and
acute lymphocytic leukemia (ALL). In collaboration with Chiara
Bonini’s team at IRCCS Ospedale San Raffaele (OSR), the AML mouse
model was developed using patient-derived primary AML blasts.
WT1-specific T cell administration inhibited tumor growth more
significantly and durably in blood, bone marrow and spleen than T
cells edited using an industry standard electroporation process.
Researchers additionally used an aggressive ALL model in
immunocompromised mice engineered to express T cell-supporting
cytokines at levels comparable to those in patients
post-conditioning regimen, or post-lymphodepletion. In the ALL
model, WT1-specific T cells also bestowed significant tumor
control.
The presentation can be found here, on the Scientific
Publications & Presentations page of Intellia’s website.
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
both producing therapeutics that permanently edit and/or correct
disease-associated genes in the human body with a single treatment
course, 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”, “we” or “our”) 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: plans to submit an investigational new
drug (“IND”) application or similar clinical trial application for
NTLA-5001, its first T cell receptor (“TCR”)-directed engineered
cell therapy development candidate for its acute myeloid leukemia
(“AML”) program in the first half of 2021; plans to advance and
complete preclinical studies and other animal studies supporting
other in vivo and ex vivo programs, including its AML program;
development of a modular platform to advance its complex genome
editing capabilities, such as gene insertion; further development
of its proprietary cell engineering process for multiple sequential
editing; 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 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;
ability to execute on its preclinical and clinical development
plans relating to NTLA-5001 and other in vivo and ex vivo programs
in view of the COVID-19 pandemic; and statements regarding the
timing of regulatory filings and clinical trial execution,
including dosing of patients, regarding its development programs;
and the potential commercial opportunities, including value and
market, for our product candidates.
Any forward-looking statements in this presentation 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 regulatory
agencies’ evaluation of regulatory filings and other information
related to its product candidates; 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, including those
that are co-developed, 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 Novartis or Regeneron or its other ex vivo
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 (“SEC”). All information in this presentation is as of
the date of the presentation, 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
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