Intellia Therapeutics Announces New, Robust Genome Editing Data for Sickle Cell Disease at the American Society of Hematology...
December 11 2017 - 7:00AM
Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading genome editing
company focused on the development of potentially curative
therapeutics using the CRISPR technology, and its collaborator,
Novartis, presented initial data from their research collaboration
on genome-edited human hematopoietic stem cells. These data showed
successful ex vivo editing of the erythroid specific enhancer of
BCL11A, a gene associated with prevention of sickle cell disease,
and the ability of these cells to stably engraft in mice while
maintaining their desired properties.
These data were presented today at the American Society of
Hematology Annual Meeting in the platform presentation session:
Hemoglobinopathies, Excluding Thalassemia – Basic and Translational
Science: Sickle Cell Disease – Hematopoiesis and Fetal Hemoglobin
Augmentation. In the presented studies, the companies:
- Achieved approximately 80-95 percent target site modification
in human hematopoietic stem and progenitor CD34+ cells following
electroporation of ribonucleoprotein (RNP) composed of Cas9 and a
guide RNA (gRNA), selected for efficacy and potency;
- Demonstrated an approximately 40 percent reduction in BCL11A
mRNA with a corresponding two-fold increase in γ-globin transcript
and 30-40 percent more fetal hemoglobin-positive cells above
background. Similar decreases in BCL11A mRNA and increases in
γ-globin transcipt were observed when sickle cell disease-derived
cells from patient donors were edited;
- Achieved engraftment over 16 weeks following transplantation of
edited human bone marrow CD34+ cells into immune compromised mice,
while maintaining editing levels in engrafted cells; and
- Observed no off-target events in CD34+ cells edited with the
selected gRNA, as measured by targeted next generation sequencing
of sites identified through in silico prediction and based on an
unbiased, genome-wide, oligo-insertion detection method.
“We are pleased to be reporting data from studies generated
through Intellia’s collaboration with Novartis, demonstrating
successful ex vivo CRISPR/Cas9 editing in hematopoietic stem
cells,” said John Leonard, M.D., executive vice president, Research
& Development, Intellia Therapeutics. “These results are
significant as we have shown high levels of editing as well as
increased production of fetal hemoglobin to clinically relevant
levels, which could potentially ameliorate sickle cell disease in
affected patients. We are very encouraged to present this progress
given that sickle cell disease is a serious condition that
currently has limited treatment options.”
About Sickle Cell Disease
Sickle cell disease is a life-threatening, hereditary disorder
that impacts approximately 30 million people worldwide. The disease
results from a single amino acid change in the β-globin gene, which
causes polymerization of hemoglobin and the deformation of red
blood cells, leading to vaso-occlusion, severe pain crisis and
multi-organ dysfunction. The average life expectancy in
the developed world is 40 to 60 years. About 80 percent of
sickle cell disease cases are believed to occur in sub-Saharan
Africa.
About Intellia Therapeutics
Intellia Therapeutics is a leading genome editing company
focused on the development of 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. Our combination of deep scientific, technical and
clinical development experience, along with our leading
intellectual property portfolio, puts us 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; Follow us
on Twitter @intelliatweets.
Forward-Looking
Statements
This press release contains “forward-looking statements” within
the meaning of the Private Securities Litigation Reform Act of
1995. Forward-looking statement in this press release include, but
are not limited to, statements regarding the successful development
of CRISPR/Cas9-based therapies to treat sickle cell disease or any
other disease; the translation of the results from animal models to
human therapies; the successful development of gRNAs with no
off-target events; and the Company’s or its collaborator’s ability
and intention to develop, seek regulatory approval for, and
commercialize therapies to treat sickle cell disease using
CRISPR/Cas9. Any forward-looking statements in this press
release are uncertain, based on management's current expectations
of future events, occurrences, actions and plans, and subject to
various 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. The Company may not
actually execute or obtain the results from the plans, goals,
efforts or opportunities disclosed in these forward-looking
statements, and you should not place undue reliance on these
forward-looking statements. Actual results or events could
differ materially from the plans, goals, efforts or opportunities
disclosed in these forward-looking statements as a result of
various factors including: uncertainties inherent in the
implementation and execution of preclinical studies and clinical
trials, and preclinical and clinical development of the Company’s
or its collaborator’s products candidates; availability and timing
of results from preclinical studies and clinical trials; whether
interim results from a preclinical trial will be predictive of the
final results of the preclinical or clinical trials or the results
of future trials; expectations and requirements for regulatory
approvals to conduct trials or to market products; and availability
of funding sufficient for the Company’s or its collaborator’s
foreseeable and unforeseeable operating expenses and capital
expenditure requirements. For a discussion of risks and
uncertainties, and other important factors, any of which could
cause our actual results to differ from those contained in the
forward-looking statements, see the section entitled "Risk Factors"
in our most recent quarterly report on Form 10-Q and in our
Prospectus filed with the Securities and Exchange Commission,
as well as discussions of potential risks, uncertainties, and other
important factors in our other filings with the Securities and
Exchange Commission. All information and forward-looking statements
in this press release are as of the date of the release,
and Intellia Therapeutics undertakes no duty to update this
information, whether because of new information, future events or
otherwise, unless required by law.
Media Contact:Jennifer Mound Smoter Senior Vice
President, External Affairs & Communications +1 857-706-1071
jenn.smoter@intelliatx.com |
Investor Contact:Lindsey TrickettVice President,
Investor Relations+1 857-285-6211
lindsey.trickett@intelliatx.com |
Intellia Therapeutics (NASDAQ:NTLA)
Historical Stock Chart
From Jun 2024 to Jul 2024
Intellia Therapeutics (NASDAQ:NTLA)
Historical Stock Chart
From Jul 2023 to Jul 2024