FDA Accepts Investigational New Drug Application for CRISPR/Cas9-Based Sickle Cell Disease Therapeutic Candidate Developed Un...
31 Mars 2020 - 1:30PM
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 the U.S. Food and Drug Administration
(FDA) has accepted the Investigational New Drug (IND) application
submitted by its collaborator, Novartis, for a CRISPR/Cas9-based
engineered cell therapy for the treatment of sickle cell disease
(SCD).
This Phase 1/2 clinical trial will begin investigating OTQ923 in
adult patients with severe complications of SCD. OTQ923 is a SCD
treatment based on genome editing of hematopoietic stem cells
(HSCs), using CRISPR/Cas9 RNA guides identified through Intellia’s
cell therapy research collaboration with Novartis. This therapeutic
approach results in highly targeted editing of the HSC’s DNA to
induce fetal hemoglobin (HbF) expression. The edited cells are
returned to the patient, where the expression of HbF is expected to
reduce the deleterious effects of sickle hemoglobin (HbS).
Novartis’ IND application triggered a milestone payment to
Intellia, and the company is eligible to receive additional
downstream success-based milestones and royalties.
“We are pleased to have worked alongside our colleagues at
Novartis to achieve this important milestone, which moves this
CRISPR/Cas9-based engineered cell therapy into the clinic, with the
potential to significantly impact the lives of patients who suffer
from sickle cell disease,” said Intellia Chief Operating Officer
and Executive Vice President Andrew Schiermeier, Ph.D. “Our
research with Novartis over the past five years has laid the
groundwork for the development of next-generation CRISPR/Cas9-based
cell therapies for patients. Intellia looks forward to Novartis’
efforts to advance other targets that were selected to develop as
additional CRISPR/Cas9-based cell therapy products.”
About Intellia’s Engineered Cell Therapy
Programs
From December 2014 through December 2019, Intellia and Novartis
jointly researched CRISPR/Cas9-based cell therapies in various cell
types, including certain stem cells and T cells. In parallel with
its ex vivo collaboration with Novartis, Intellia has been
advancing its wholly owned ex vivo pipeline of immuno-oncology and
autoimmune cell therapies. Intellia’s proprietary ex vivo programs
include its acute myeloid leukemia (AML) program utilizing
transgenic T cell receptors (TCRs) against Wilms’ Tumor 1 (WT1), a
target identified in collaboration with IRCCS Ospedale San Raffaele
(OSR). Intellia plans to submit an IND application for NTLA-5001,
the company’s development candidate for the treatment of AML, in
the first half of 2021. View Intellia’s programs pipeline for more
information.
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 beliefs and
expectations regarding its: planned submission of 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 for its
other in vivo and ex vivo programs; 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 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 optimize the
impact of its collaborations on its development programs, including
but not limited to its collaborations with Novartis and OSR;
statements regarding the timing of regulatory filings regarding its
development programs; use of capital, expenses, future accumulated
deficit and other 2019 financial results or in the future; and
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 Novartis 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. 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:Jennifer Mound SmoterSenior Vice
President
External Affairs &
Communications
+1 857-706-1071
jenn.smoter@intelliatx.com
Lynnea OlivarezDirectorExternal Affairs & Communications+1
956-330-1917lynnea.olivarez@intelliatx.com
Investors:Lina LiAssociate DirectorInvestor
Relations+1 857-706-1612lina.li@intelliatx.com
Intellia Therapeutics (NASDAQ:NTLA)
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