CRISPR Therapeutics and Vertex Announce FDA Has Lifted the Clinical Hold on the Investigational New Drug Application for CTX0...
October 10 2018 - 4:01PM
CRISPR Therapeutics (NASDAQ:CRSP), a biopharmaceutical company
focused on creating transformative gene-based medicines for serious
diseases, and Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX)
today announced that the U.S. Food and Drug Administration (FDA)
has lifted the clinical hold and accepted the Investigational New
Drug application (IND) for CTX001 for the treatment of sickle cell
disease (SCD). CTX001 is an investigational, autologous,
gene-edited hematopoietic stem cell therapy for patients suffering
from severe hemoglobinopathies.
In addition to the acceptance of the IND
announced today, CRISPR and Vertex previously announced that they
had obtained approvals of Clinical Trial Applications for CTX001 in
multiple countries outside the U.S. for both β-thalassemia and SCD.
The companies remain on track to initiate a Phase 1/2 clinical
study in SCD by the end of 2018 and are currently enrolling
patients with transfusion dependent β-thalassemia in a Phase 1/2
trial in β-thalassemia in Europe.
About CTX001CTX001 is an
investigational ex vivo CRISPR gene-edited therapy for patients
suffering from β-thalassemia or 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.
CTX001 is being developed under a co-development
and co-commercialization agreement between CRISPR Therapeutics and
Vertex. The companies have obtained approvals of Clinical Trial
Applications (CTA) in multiple countries for both β-thalassemia and
sickle cell disease (SCD).
About the CRISPR-Vertex
Collaboration CRISPR 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 platform. CRISPR/Cas9 is a
revolutionary gene editing technology that allows for precise,
directed changes to genomic DNA. CRISPR Therapeutics has
established a portfolio of therapeutic programs across a broad
range of disease areas including hemoglobinopathies, oncology,
regenerative medicine and rare diseases. To accelerate and expand
its efforts, CRISPR Therapeutics has established strategic
collaborations with leading companies including Bayer AG, Vertex
Pharmaceuticals and ViaCyte, Inc. 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, and business offices in London, United
Kingdom. For more information, please visit www.crisprtx.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, any
approvals thereof and timing of commencement of clinical trials,
the intellectual property coverage and positions of CRISPR
Therapeutics, its licensors and third parties, the sufficiency of
CRISPR Therapeutics’ 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 CRISPR Therapeutics
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 CRISPR
Therapeutics’ 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 CRISPR Therapeutics’
most recent annual report on Form 10-K, and in any other subsequent
filings made by CRISPR Therapeutics 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. CRISPR Therapeutics
disclaims any obligation or undertaking to update or revise any
forward-looking statements contained in this press release, other
than to the extent required by law.
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 eight years in a row.
For additional information and the latest
updates from the company, please visit www.vrtx.com.
Vertex 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
information provided regarding the status of, and expectations with
respect to, the CTX001 clinical development program. 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
that the development of CTX001 may not proceed 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)
CRISPR Investor Contact:Susan
Kimsusan.kim@crisprtx.com
CRISPR Media Contact:Jennifer PaganelliWCG on
behalf of CRISPR347-658-8290jpaganelli@wcgworld.com
Vertex Pharmaceuticals
IncorporatedInvestors:Michael Partridge,
+1 617-341-6108orEric Rojas, +1 617-961-7205orZach Barber, +1
617-341-6470or
Media:Heather Nichols,
617-341-6992mediainfo@vrtx.com
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