CRISPR Therapeutics Announces Completion of FDA Advisory Committee Meeting for Exagamglogene Autotemcel (exa-cel) for Severe Sickle Cell Disease
October 31 2023 - 5:53PM
CRISPR Therapeutics (Nasdaq: CRSP) today announced the
completion of the U.S. Food and Drug
Administration’s (FDA) Cellular, Tissue, and Gene
Therapies Advisory Committee meeting for exagamglogene
autotemcel (exa-cel) for the treatment of SCD in people ages 12 and
older with recurrent vaso-occlusive crises (VOCs). Exa-cel is the
first potential therapy to emerge from a strategic partnership
between CRISPR Therapeutics and Vertex Pharmaceuticals.
If approved, exa-cel could be the first genetic
therapy available to approximately twenty thousand people with
severe SCD in the U.S. The FDA granted priority review for exa-cel
in the treatment of people with SCD and assigned a Prescription
Drug User Fee Act (PDUFA) action date of December 8, 2023.
Exa-cel’s Biologics License Application (BLA) for
transfusion-dependent beta-thalassemia (TDT) was assigned a PDUFA
date of March 30, 2024.
About the CRISPR Therapeutics and 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. Exa-cel
represents the first potential treatment to emerge from the joint
research program. Under an amended collaboration agreement, Vertex
now leads global development, manufacturing and commercialization
of exa-cel and splits program costs and profits worldwide 60/40
with CRISPR Therapeutics.
(CRSP-GEN)
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 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, 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 in
Boston, Massachusetts and San Francisco, California, and business
offices in London, United Kingdom. For more information, please
visit www.crisprtx.com.
CRISPR THERAPEUTICS® word mark and design are
trademarks and registered trademarks of CRISPR Therapeutics AG. All
other trademarks and registered trademarks are the property of
their respective owners. CRISPR Therapeutics
Forward-Looking Statement
This 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) exa-cel’s potential for approval in TDT
and SCD, including accelerated approval for SCD in the U.S., as
well as the timing of any such approval by the FDA and that exa-cel
could be the first genetic therapy available to eligible patients
with SCD in the U.S. if approved; (ii) potential benefits of
exa-cel for patients; (iii) timelines for and expectations
regarding additional regulatory agency decisions; (iv) the benefits
of its collaboration with Vertex; and (v) 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, existing and
prospective investors are cautioned that forward-looking statements
are inherently uncertain, are neither promises nor guarantees and
not to place undue reliance on such statements, which speak only as
of the date they are made. 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, that: the
efficacy and safety results from ongoing clinical trials of exa-cel
will not continue or be repeated in ongoing or planned clinical
trials or may not support regulatory submissions; the FDA or other
regulatory authorities may not approve exa-cel on a timely basis or
at all; adequate pricing or reimbursement may not be secured to
support continued development or commercialization of exa-cel
following regulatory approval; future competitive or other market
factors may adversely affect the commercial potential for exa-cel;
CRISPR Therapeutics may not realize the potential benefits of its
collaboration with Vertex; uncertainties regarding the intellectual
property protection for CRISPR Therapeutics’ technology and
intellectual property belonging to third parties; and those risks
and uncertainties described under the heading “Risk Factors” in
CRISPR Therapeutics’ most recent annual report on Form 10-K,
quarterly report on Form 10-Q, 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. 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.
CRISPR Therapeutics:
Investors:Susan
Kim+1-617-307-7503susan.kim@crisprtx.com
Media:Rachel
Eides+1-617-315-4493rachel.eides@crisprtx.com
Source: CRISPR Therapeutics
CRISPR Therapeutics (NASDAQ:CRSP)
Historical Stock Chart
From Apr 2024 to May 2024
CRISPR Therapeutics (NASDAQ:CRSP)
Historical Stock Chart
From May 2023 to May 2024