CRISPR Therapeutics (Nasdaq: CRSP) and Vertex Pharmaceuticals
Incorporated (Nasdaq: VRTX) today announced that the U.S. Food and
Drug Administration (FDA) granted Regenerative Medicine Advanced
Therapy (RMAT) designation to CTX001, an investigational,
autologous, gene-edited hematopoietic stem cell therapy, for the
treatment of severe sickle cell disease (SCD) and
transfusion-dependent beta thalassemia (TDT).
“RMAT designation is another important regulatory milestone for
CTX001 and underscores the transformative potential of a
CRISPR-based therapy for patients with severe hemoglobinopathies,”
said Samarth Kulkarni, Ph.D., Chief Executive Officer of CRISPR
Therapeutics. “We expect to share additional clinical data on
CTX001 in medical and scientific forums this year as we continue to
work closely with global regulatory agencies to expedite the
clinical development of CTX001.”
“The first clinical data announced for CTX001 late last year
represented a key advancement in our efforts to bring CRISPR-based
therapies to people with beta thalassemia and sickle cell disease
and demonstrate the curative potential of this therapy,” said
Bastiano Sanna, Ph.D., Executive Vice President and Chief of Cell
and Genetic Therapies at Vertex. “We are encouraged by these recent
regulatory designations from the FDA and EMA, which speak to the
potential impact this therapy could have for patients.”
Established under the 21st Century Cures Act, RMAT designation
is a dedicated program designed to expedite the drug development
and review processes for promising pipeline products, including
genetic therapies. A regenerative medicine therapy is eligible for
RMAT designation if it is intended to treat, modify, reverse or
cure a serious or life-threatening disease or condition, and
preliminary clinical evidence indicates that the drug or therapy
has the potential to address unmet medical needs for such disease
or condition. Similar to Breakthrough Therapy designation, RMAT
designation provides the benefits of intensive FDA guidance on
efficient drug development, including the ability for early
interactions with FDA to discuss surrogate or intermediate
endpoints, potential ways to support accelerated approval and
satisfy post-approval requirements, potential priority review of
the biologics license application (BLA) and other opportunities to
expedite development and review.
In addition to RMAT designation, CTX001 has received Orphan Drug
Designation from the U.S. FDA for TDT and from the European
Commission for TDT and SCD. CTX001 also has Fast Track Designation
from the U.S. FDA for both TDT and SCD.
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) and
discussions with regulatory authorities related to product
candidates under development by CRISPR Therapeutics and its
collaborators, including expectations regarding the benefits of
RMAT designation; (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, the
information provided regarding the status of, and expectations with
respect to, the CTX001 clinical development program and related
global regulatory approvals, and expectations regarding the RMAT
designation. 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.: +1 617-341-6992or Heather Nichols: +1
617-961-0534or International: +44 20 3204 5275
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