CRISPR Therapeutics (Nasdaq: CRSP) and Vertex Pharmaceuticals
Incorporated (Nasdaq: VRTX) today announced that new data from two
ongoing Phase 1/2 clinical trials of the CRISPR/Cas9 gene-editing
therapy CTX001 in severe hemoglobinopathies have been accepted for
an oral presentation at the EHA Congress, which will take place
virtually from June 11-14, 2020.
An abstract posted online today includes 12 months of follow-up
data for the first patient treated in the ongoing Phase 1/2
CLIMB-111 trial in transfusion-dependent beta thalassemia (TDT) and
6 months of follow-up data for the first patient treated in the
ongoing Phase 1/2 CLIMB-121 trial in severe sickle cell disease
(SCD). Updated data will be presented at EHA, including longer
duration follow-up data for the first two patients treated in these
trials and initial data for the second patient treated in the
CLIMB-111 trial.
The accepted abstract is now available on the EHA conference
website: https://ehaweb.org/congress/eha25/key-information-2/.
Abstract Title: Initial Safety and Efficacy Results With a
Single Dose of Autologous CRISPR-Cas9 Modified CD34+ Hematopoietic
Stem and Progenitor Cells in Transfusion-Dependent β-Thalassemia
and Sickle Cell DiseaseSession Title: Immunotherapy -
ClinicalAbstract Code: S280
About the Phase 1/2 Study in Transfusion-Dependent Beta
Thalassemia The ongoing Phase 1/2 open-label trial,
CLIMB-Thal-111, is designed to assess the safety and efficacy of a
single dose of CTX001 in patients ages 18 to 35 with TDT. The study
will enroll up to 45 patients and follow patients for approximately
two years after infusion. Each patient will be asked to participate
in a long-term follow-up study.
About the Phase 1/2 Study in Sickle Cell
Disease The ongoing Phase 1/2 open-label trial,
CLIMB-SCD-121, is designed to assess the safety and efficacy of a
single dose of CTX001 in patients ages 18 to 35 with severe SCD.
The study will enroll up to 45 patients and follow patients for
approximately two years after infusion. Each patient will be asked
to participate in a long-term follow-up study.
About CTX001CTX001 is an investigational ex vivo CRISPR
gene-edited therapy that is being evaluated for patients suffering
from TDT or severe SCD 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 TDT patients and painful and debilitating sickle
crises for SCD patients. CTX001 is the most advanced gene-editing
approach in development for beta thalassemia and SCD.
CTX001 is
being developed under a co-development and co-commercialization
agreement between CRISPR Therapeutics and Vertex.
About the CRISPR-Vertex
CollaborationCRISPR
Therapeutics and Vertex entered into a strategic research
collaboration in 2015 focused on the use of CRISPR/Cas9 to discover
and develop potential new treatments aimed at the underlying
genetic causes of human disease. CTX001 represents the first
treatment to emerge from the joint research program. CRISPR
Therapeutics and Vertex will jointly develop and commercialize
CTX001 and 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
partnerships with leading companies including Bayer, 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 San Francisco,
California and London, United Kingdom. For more information, please
visit www.crisprtx.com.
CRISPR Forward-Looking
StatementThis press release may contain a number of
“forward-looking statements” within the meaning of the Private
Securities Litigation Reform Act of 1995, as amended, including
statements regarding CRISPR Therapeutics’ expectations about any or
all of the following: (i) the status of clinical trials (including,
without limitation, the expected timing of data releases) related
to product candidates under development by CRISPR Therapeutics and
its collaborators, including expectations regarding the data that
is expected to be presented at the European Hematology
Association’s upcoming congress; (ii) the expected benefits of
CRISPR Therapeutics’ collaborations; and (iii) the therapeutic
value, development, and commercial potential of CRISPR/Cas9 gene
editing technologies and therapies. Without limiting the foregoing,
the words “believes,” “anticipates,” “plans,” “expects” and similar
expressions are intended to identify forward-looking statements.
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, 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: the potential impacts due to the coronavirus
pandemic, such as the timing and progress of clinical trials; the
potential for initial and preliminary data from any clinical trial
and initial data from a limited number of patients (as is the case
with CTX001 at this time) not to be indicative of final trial
results; the potential that CTX001 clinical trial results may not
be favorable; that future competitive or other market factors may
adversely affect the commercial potential for CTX001; uncertainties
regarding the intellectual property protection for CRISPR
Therapeutics’ technology and intellectual property belonging to
third parties, and the outcome of proceedings (such as an
interference, an opposition or a similar proceeding) involving all
or any portion of such intellectual property; 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, 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 diseases.
The company has multiple approved medicines that treat the
underlying cause of cystic fibrosis (CF) — a rare, life-threatening
genetic disease — and has several ongoing clinical and research
programs in CF. Beyond CF, Vertex has a robust pipeline of
investigational small molecule medicines in other serious diseases
where it has deep insight into causal human biology, including
pain, alpha-1 antitrypsin deficiency and APOL1-mediated kidney
diseases. In addition, Vertex has a rapidly expanding pipeline of
genetic and cell therapies for diseases such as sickle cell
disease, beta thalassemia, Duchenne muscular dystrophy and type 1
diabetes mellitus.
Founded in 1989 in Cambridge, Mass., Vertex's
global headquarters is now located in Boston's Innovation District
and its international headquarters is in London, UK. Additionally,
the company has research and development sites and commercial
offices in North America, Europe, Australia and Latin America.
Vertex is consistently recognized as one of the industry's top
places to work, including 10 consecutive years on Science
magazine's Top Employers list and top five on the 2019 Best
Employers for Diversity list by Forbes. For company updates and to
learn more about Vertex's history of innovation, visit
www.vrtx.com/ or follow us on Facebook, Twitter, LinkedIn, YouTube
and Instagram.
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,
information regarding the data that is expected to be presented at
the European Hematology Association (EHA)’s upcoming Congress.
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 the development of CTX001 may not proceed
or support registration 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 Therapeutics Investor Contact:Susan Kim,
+1 617-307-7503susan.kim@crisprtx.com
CRISPR Therapeutics Media Contact:Rachel
EidesWCG on behalf of CRISPR+1 617-337-4167 reides@wcgworld.com
Vertex Pharmaceuticals Incorporated
Investors: Michael Partridge, +1 617-341-6108 or
Zach Barber, +1 617-341-6470 or Brenda Eustace, +1 617-341-6187
Media: mediainfo@vrtx.com orU.S.: +1 617-341-6992
orHeather Nichols: +1 617-839-3607or International: +44 20 3204
5275
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