CRISPR Therapeutics Announces Oral Presentation of New Data on CTX001, a CRISPR Gene-Edited Therapy for β-Thalassemia and Si...
December 10 2017 - 7:45AM
CRISPR Therapeutics (NASDAQ:CRSP) today announced the presentation
of new data on CTX001, an investigational CRISPR gene-edited
therapy for patients suffering from β-thalassemia and sickle cell
disease. The data were discussed in an oral presentation at the
American Society of Hematology (ASH) Annual Meeting on Sunday,
December 10th, 2017.
“With CTX001, we are able to efficiently edit
hematopoietic stem cells to achieve high levels of fetal
hemoglobin, a protein that is known to significantly reduce or even
eliminate the transfusion dependence of β-thalassemia patients and
the frequency of painful and debilitating sickle crises in sickle
cell patients. These data strongly support the advancement of
CTX001 into clinical trials in patients,” commented Dr. Tony Ho,
MD, Head of R&D of CRISPR Therapeutics.
CRISPR Therapeutics has filed a Clinical Trial
Application for CTX001, and is planning to start a Phase 1/2 trial
in β-thalassemia in Europe in 2018.
Key Data from ASH Session
112
Data presented at ASH demonstrate that CRISPR
Therapeutics’ proprietary CRISPR gene-editing approach results in
high editing efficiency, with >90% of the hematopoietic stem
cells edited at the target site. A vast majority of these cells are
edited on both copies of the gene, which leads to expression levels
of fetal hemoglobin of 40%, well above the level believed to be
sufficient to ameliorate symptoms in patients with β-thalassemia
and sickle cell disease.
CRISPR conducted extensive genome-wide
off-target assessment including detailed analyses at over 6,000
sites, which showed no off-target editing. Full toxicology
analysis also demonstrated that the CRISPR gene-editing had no
adverse impact on engraftment of the hematopoietic stem cells and
no other safety signals.
These results were presented in an oral session
by Dr. Bill Lundberg, Chief Scientific Officer of CRISPR in a
session entitled CRISPR/Cas9 Genome Editing to Treat Sickle Cell
Disease and β-Thalassemia: Re-Creating Genetic Variants to
Upregulate Fetal Hemoglobin Appear Well-Tolerated, Effective and
Durable.
Slides from the ASH presentation are available
under the Investors and Media tab on the CRISPR Therapeutics
website.
About CTX001
CTX001 is an investigational ex vivo CRISPR
gene-edited therapy for patients suffering from β-thalassemia and
sickle cell disease in which a patient’s hematopoietic stem cells
are engineered to produce high levels of fetal hemoglobin (HbF;
hemoglobin F) in red blood cells. HbF is a form of the oxygen
carrying hemoglobin that is naturally present at birth, and is then
replaced by the adult form of hemoglobin. The elevation of HbF by
CTX001 has the potential to alleviate transfusion-requirements for
β-thalassemia patients and painful and debilitating sickle crises
for sickle cell patients.
About the CRISPR-Vertex
Collaboration
CTX001 is the first CRISPR/Cas9-based treatment
to advance from a research program jointly conducted by CRISPR
Therapeutics and Vertex Pharmaceuticals under the companies’
collaboration aimed at the discovery and development of new gene
editing treatments that use the CRISPR/Cas9 technology. Under the
agreement, Vertex has exclusive rights to license up to six new
CRISPR/Cas9-based treatments that emerge from the
collaboration.
About CRISPR
Therapeutics
CRISPR Therapeutics is a leading
gene-editing company focused on developing transformative
gene-based medicines for serious diseases using its proprietary
CRISPR/Cas9 gene-editing platform. CRISPR/Cas9 is a revolutionary
technology that allows for precise, directed changes to genomic
DNA. The company's multi-disciplinary team of world-class
researchers and drug developers is working to translate this
technology into breakthrough human therapeutics in a number of
serious diseases. Additionally, CRISPR Therapeutics has
established strategic collaborations with Bayer
AG and Vertex Pharmaceuticals to develop
CRISPR-based therapeutics in diseases with high unmet need. The
foundational CRISPR/Cas9 patent estate for human therapeutic use
was licensed from the company's scientific founder Emmanuelle
Charpentier, Ph.D. CRISPR Therapeutics AG is
headquartered in Zug, Switzerland, with its wholly-owned U.S.
subsidiary, CRISPR Therapeutics, Inc., and R&D operations
based in Cambridge, Massachusetts. For more information,
please visit http://www.crisprtx.com.
CRISPR Forward-Looking
Statement
Certain statements set forth in this press
release constitute “forward-looking statements” within the meaning
of the Private Securities Litigation Reform Act of 1995, as
amended, including, but not limited to, statements concerning: the
timing of filing of clinical trial applications and INDs and timing
of commencement of clinical trials, the intellectual property
coverage and positions of the Company, its licensors and third
parties, the sufficiency of the Company’s cash resources and the
therapeutic value, development, and commercial potential of
CRISPR/Cas-9 gene editing technologies and therapies. You are
cautioned that forward-looking statements are inherently uncertain.
Although the Company believes that such statements are based on
reasonable assumptions within the bounds of its knowledge of its
business and operations, the forward-looking statements are neither
promises nor guarantees and they are necessarily subject to a high
degree of uncertainty and risk. Actual performance and results may
differ materially from those projected or suggested in the
forward-looking statements due to various risks and uncertainties.
These risks and uncertainties include, among others: uncertainties
regarding the intellectual property protection for our technology
and intellectual property belonging to third parties; uncertainties
inherent in the initiation and completion of preclinical studies
for the Company’s product candidates; availability and timing of
results from preclinical studies; whether results from a
preclinical trial will be predictive of future results of the
future trials; expectations for regulatory approvals to conduct
trials or to market products; and those risks and uncertainties
described under the heading “Risk Factors” in the Company’s most
recent annual report on Form 10-K, and in any other subsequent
filings made by the Company with the U.S. Securities and
Exchange Commission (SEC), which are available on the SEC’s
website at www.sec.gov. Existing and prospective investors are
cautioned not to place undue reliance on these forward-looking
statements, which speak only as of the date they are made.
CONTACTS
CRISPR Therapeutics
Investors:Chris Erdman
617-307-7227Chris.erdman@crisprtx.comorChris Brinzey Westwicke
Partners339-970-2843chris.brinzey@westwicke.comorMedia:Jennifer
Paganelli WCG for CRISPR 347-658-8290 jpaganelli@wcgworld.com
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