-Vertex selects CTX001 as first gene edited
treatment to be developed as part of collaboration with CRISPR
Therapeutics-
-Clinical Trial Application for CTX001
submitted in Europe to support initiation of Phase 1/2 clinical
study in β-thalassemia in 2018-
-Preclinical data for CTX001 presented this
week at the American Society for Hematology Annual Meeting-
Vertex Pharmaceuticals Incorporated (NASDAQ: VRTX) and CRISPR
Therapeutics AG (NASDAQ: CRSP) today announced that the companies
will co-develop and co-commercialize CTX001, an investigational
gene editing treatment, as part of the companies’ previously
announced collaboration aimed at the discovery and development of
new gene editing treatments that use the CRISPR/Cas9 technology.
CTX001 represents the first gene-based treatment that Vertex
exclusively licensed from CRISPR Therapeutics as part of the
collaboration. For CTX001, CRISPR and Vertex will equally share all
research and development costs and profits worldwide. A
Clinical Trial Application was submitted earlier this month for
CTX001 to support the initiation of a Phase 1/2 trial in
β-thalassemia in 2018 in Europe, and an Investigational New Drug
(IND) Application is planned for submission in 2018 to support the
initiation of a Phase 1/2 trial in sickle cell disease in the U.S.
Preclinical data presented for CTX001 at the American Society for
Hematology on December 10, 2017 showed clinically relevant
increases in fetal hemoglobin and a high editing rate that support
the advancement of CTX001 into the planned trials in β-thalassemia
and sickle cell disease in 2018.
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“Over the past two years, we’ve made significant progress with
CRISPR Therapeutics on the discovery and preclinical development of
multiple CRISPR/Cas9-based treatments, and we’re pleased to select
CTX001 as the first of these treatments to move into clinical
development as part of our collaboration,” said David Altshuler,
M.D., Ph.D., Vertex’s Executive Vice President, Global Research and
Chief Scientific Officer. “The addition of CTX001 to our clinical
development pipeline provides us with a near-term opportunity to
generate the first proof-of-concept clinical data for a
CRISPR/Cas9-based medicine in two genetic diseases that are highly
aligned with our research strategy.”
“The submission of a Clinical Trial Application for CTX001 in
Europe, supported by the robust data presented at the recent ASH
Annual Meeting, reflect the advances we have achieved in
translating the potential of CRISPR/Cas9 science into
transformative therapies. We now look forward to working closely
with Vertex as we initiate clinical trials next year,” commented
Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR
Therapeutics. “The study of CTX001 in β-thalassemia will be the
first company-sponsored clinical trial of a CRISPR-based therapy
and is a major step forward for both the treatment of certain
inherited blood diseases and for our collaboration with
Vertex.”
Clinical Development Plans for CTX001CRISPR Therapeutics
and Vertex will co-develop and co-commercialize CTX001 for the
treatment of hemoglobinopathies, including β-thalassemia and sickle
cell disease. A Phase 1/2 trial of CTX001 is expected to begin in
2018 in Europe and will be designed to assess the safety and
efficacy of CTX001 in adult transfusion dependent β-thalassemia
patients. The companies also plan to file an IND Application for
CTX001 with the United States Food and Drug Administration to
support the initiation of a Phase 1/2 trial in sickle cell disease
in 2018 in the U.S. Additional details on the trial designs will be
provided upon study initiation.
About CTX001 and Recent Data Presented at the American
Society for Hematology (ASH) Annual MeetingCTX001 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.
On December 10, 2017, CRISPR Therapeutics presented preclinical
data at the 2017 ASH Annual Meeting that showed greater than 90%
editing of hematopoietic stem cells at the target site, leading to
clinically relevant increases in fetal hemoglobin. These data
support the advancement of CTX001 into the planned trials in
β-thalassemia and sickle cell disease in 2018.
About the CRISPR-Vertex CollaborationCRISPR and Vertex
entered into a strategic research collaboration in 2015 aimed at
the discovery and development of gene editing treatments using the
CRISPR/Cas9 technology to correct defects in specific gene targets
known to cause or contribute to particular diseases. Vertex has
exclusive rights to license up to six new CRISPR/Cas9-based
treatments that emerge from the collaboration, and CTX001
represents the first treatment to emerge from the joint research
program. For CTX001, CRISPR and Vertex will equally share all
research and development costs and profits worldwide.
About CRISPR TherapeuticsCRISPR 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.
About VertexVertex is a global biotechnology company that
invests in scientific innovation to create transformative medicines
for people with serious and life-threatening diseases. In addition
to clinical development programs in CF, Vertex has more than a
dozen ongoing research programs focused on the underlying
mechanisms of other serious diseases.
Founded in 1989 in Cambridge, Mass., Vertex's headquarters
is now located in Boston's Innovation District. Today, the
company has research and development sites and commercial offices
in the United
States, Europe, Canada and Australia. Vertex is
consistently recognized as one of the industry's top places to
work, including being named to Science magazine's Top
Employers in the life sciences ranking for seven years in a row.
For additional information and the latest updates from the company,
please visit www.vrtx.com.
CRISPR Forward-Looking StatementCertain 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/Cas9 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.
Vertex's Special Note Regarding Forward-looking
StatementsThis press release contains forward-looking
statements as defined in the Private Securities Litigation Reform
Act of 1995, including, without limitation, the statements in the
second and third paragraphs and statements regarding the planned
clinical development timeline, including submission of an IND to
the FDA. While Vertex believes the forward-looking statements
contained in this press release are accurate, these forward-looking
statements represent the company's beliefs only as of the date of
this press release, and there are a number of factors that could
cause actual events or results to differ materially from those
indicated by such forward-looking statements. Those risks and
uncertainties include, among other things, that data from the
company's development programs may not support registration or
further development of its compounds due to safety, efficacy or
other reasons and other risks listed under Risk Factors in Vertex's
annual report and quarterly reports filed with the Securities and
Exchange Commission and available through the company's website at
www.vrtx.com. Vertex disclaims any obligation to update the
information contained in this press release as new information
becomes available.
(VRTX-GEN)
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CRISPR TherapeuticsInvestors:Chris Erdman, +1
617-307-7227Chris.erdman@crisprtx.comorWestwicke PartnersChris
Brinzey, +1 339-970-2843chris.brinzey@westwicke.comorMedia:WCG for
CRISPRJennifer Paganelli, +1
347-658-8290jpaganelli@wcgworld.comorVertexInvestors:Michael
Partridge, +1 617-341-6108orEric Rojas, +1 617-961-7205orZach
Barber, +1 617-341-6470orMedia:Heather Nichols, +1
617-961-5093mediainfo@vrtx.com
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