Sarepta Therapeutics and Genethon Announce a Gene Therapy Research Collaboration for the Treatment of Duchenne Muscular Dystr...
June 21 2017 - 7:00AM
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a U.S. commercial-stage
biopharmaceutical company focused on the discovery and development
of unique RNA-targeted therapeutics for the treatment of rare
neuromuscular diseases, and Genethon, a non-profit R&D
organization dedicated to the development of biotherapies for
orphan genetic diseases from research to clinical validation, have
signed a gene therapy research collaboration to jointly develop
treatments for Duchenne muscular dystrophy (DMD). Genethon’s
micro-dystrophin gene therapy approach can target the majority of
patients with DMD. Genethon has demonstrated proof-of-concept of
their micro-dystrophin program via robust gene expression in a
large animal model of DMD.
Under the terms of the collaboration,
Genethon will be responsible for the early development work.
Sarepta has the option to co-develop Genethon’s
micro-dystrophin program, which includes exclusive U.S.
commercial rights. Financial terms of the collaboration have not
been disclosed.
Genethon has made significant investment in the
development of gene therapies for neuromuscular diseases and
employs one of the largest research and clinical groups in the
world working to advance rare disease therapies. The Company’s
European-based research laboratory has a long-term commitment to
neuromuscular diseases with a central focus on DMD. In addition,
Genethon is affiliated with Europe’s largest cGMP vector
manufacturing facility, YposKesi, located in Evry (Essonne).
YposKesi employs approximately 150 experts in bio-production at its
current 54,000 square feet manufacturing facility, and plans
significant future expansion to meet the growing demand of gene
therapy products.
“Our agreement with Genethon strengthens our ongoing commitment
to patients and is aligned with our strategy of building the
industry’s most comprehensive franchise in DMD,” stated Edward
Kaye, Sarepta’s chief executive officer. “This partnership brings
together our collective experience in Duchenne drug development and
Genethon’s particular expertise in gene therapy for rare
diseases. We look forward to working with Genethon given
their knowledge, large infrastructure and state-of the-art
manufacturing capabilities to advance next generation therapies for
DMD.”
“Microdystrophin-based gene therapy is a very promising approach
with potential application to a large majority of Duchenne
patients. In order to accelerate the development of a treatment, we
are very pleased to partner with Sarepta Therapeutics, which has
demonstrated commitment and success for innovative therapies for
Duchenne muscular dystrophy. This partnership brings together the
highly complementary and synergistic expertises of Sarepta and
Genethon, to the benefit of the patients,” said Frederic Revah,
chief executive officer of Genethon.
About Sarepta
TherapeuticsSarepta Therapeutics is a U.S.
commercial-stage biopharmaceutical company focused on the discovery
and development of unique RNA-targeted therapeutics for the
treatment of rare neuromuscular diseases. The Company is primarily
focused on rapidly advancing the development of its potentially
disease-modifying Duchenne muscular dystrophy (DMD) drug
candidates. For more information, please visit www.sarepta.com.
About GenethonCreated by the
AFM-Telethon, the French Muscular Dystrophy Association (AFM),
Genethon, located in Evry, France, is a non-profit R&D
organization dedicated to the development of biotherapies for
orphan genetic diseases, from the research to clinical validation.
Genethon, is specialized in the discovery and development of gene
therapy drugs and has multiple ongoing programs at clinical,
preclinical and research stage for neuromuscular, blood, immune
system, liver and eye diseases.
Forward-Looking StatementThis press release
contains statements that are forward-looking. Any statements
contained in this press release that are not statements of
historical fact may be deemed to be forward-looking statements.
Words such as "believes," "anticipates," "plans," "expects,"
"will," "intends," "potential," "possible" and similar expressions
are intended to identify forward-looking statements. These
forward-looking statements include statements about the gene
therapy research collaboration agreement Sarepta has entered into
with Genethon, Sarepta’s option to co-develop Genethon’s
micro-dystrophin program, the potential for gene therapy to advance
next generation therapies for DMD, the alignment of the
collaboration agreement with Sarepta’s strategy to build the
industry’s most comprehensive franchise in DMD, and the potential
application of microdystrophin-based gene therapy to a large
majority of Duchenne patients.
These forward-looking statements involve risks and
uncertainties, many of which are beyond Sarepta's control. Known
risk factors include, among others: the expected benefits and
opportunities related to the collaboration agreement between the
parties may not be realized or may take longer to realize than
expected due to challenges and uncertainties inherent in product
research and development; the collaboration between Sarepta and
Genethon may not result in any viable treatments suitable for
clinical research or commercialization due to a variety of reasons
including that the results of additional research may not be
consistent with past results or may not be positive or may
otherwise fail to meet regulatory approval requirements for the
safety and efficacy of product candidates or may never become
commercialized products due to other various reasons including any
potential future inability of the parties to fulfill their
commitments and obligations under the agreement; and even if the
agreement results in commercialized products, the parties may not
achieve any significant revenues from the sale of such
products.
Any of the foregoing risks could adversely affect Sarepta's
business, results of operations and the trading price of Sarepta's
common stock. For a detailed description of risks and uncertainties
Sarepta faces, you are encouraged to review Sarepta's 2016 Annual
Report on Form 10-K and most recent Quarterly Report on Form 10-Q
for the quarter ended March 31, 2017 filed with the Securities and
Exchange Commission (SEC) as well as other SEC filings made by
Sarepta. We caution investors not to place considerable reliance on
the forward-looking statements contained in this press release.
Sarepta does not undertake any obligation to publicly update its
forward-looking statements based on events or circumstances after
the date hereof.
Internet Posting of InformationWe routinely
post information that may be important to investors in the 'For
Investors' section of our website at www.sarepta.com. We
encourage investors and potential investors to consult our website
regularly for important information about us.
Media and Investors:
Sarepta Therapeutics, Inc.
Ian Estepan, 617-274-4052
iestepan@sarepta.com
or
W2O Group
Brian Reid, 212-257-6725
breid@w2ogroup.com
and
Genethon
Stéphanie Bardon, +331-69-47-12-78
sbardon@afm-telethon.fr
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