CRISPR Therapeutics Submits First Clinical Trial Application for a CRISPR Gene-Edited Therapy, CTX001 in β-thalassemia
December 07 2017 - 7:00AM
CRISPR Therapeutics (NASDAQ:CRSP) today announced the submission of
a Clinical Trial Application (CTA) for CTX001 in β-thalassemia.
CTX001 is an investigational CRISPR gene-edited autologous
hematopoietic stem cell therapy for patients suffering from
β-thalassemia and sickle cell disease.
“CRISPR Therapeutics is pioneering a new class
of medicines with the CTA submission for CTX001 to conduct the
first company-sponsored clinical trial of a CRISPR gene-edited
therapy,” commented Samarth Kulkarni, Ph.D., Chief Executive
Officer of CRISPR Therapeutics. “We are committed to translating
the groundbreaking science of the CRISPR platform into therapies
that can fundamentally change the lives of patients suffering from
serious diseases such as β-thalassemia and sickle cell
disease.”
The Phase 1/2 trial of CTX001 is designed to
assess its safety and efficacy in adult transfusion dependent
β-thalassemia patients and is expected to begin in Europe in 2018.
CRISPR also plans to file an Investigational New Drug Application
for CTX001 to treat sickle cell disease with the United States Food
and Drug Administration in 2018.
“β-thalassemia is a devastating disease that
requires serious and chronic medical intervention,” said Tony Ho,
M.D., Head of Research and Development at CRISPR. “The efficient
and precise editing in a patient’s own blood cells using CRISPR
provides the possibility of a one-time treatment for those
suffering from β-thalassemia and sickle cell disease.”
About CTX001
CTX001 is an investigational 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 617-307-7227
Chris.erdman@crisprtx.com orChris Brinzey Westwicke
Partners339-970-2843chris.brinzey@westwicke.com orMedia:Jennifer
Paganelli WCG for CRISPR 347-658-8290
jpaganelli@wcgworld.com
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