Sarepta Therapeutics Enters into License Agreement with Nationwide Children’s Hospital for Galgt2 Gene Therapy Program
January 10 2017 - 8:30AM
Business Wire
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a commercial
stage developer of innovative RNA-targeted therapeutics, today
announced it has entered an exclusive license agreement with
Nationwide Children’s Hospital, for their Galgt2 gene therapy
program developed by researcher Dr. Paul Martin, Ph.D.
“We are taking a multi-front approach in the battle against
Duchenne muscular dystrophy,” said Edward Kaye, Sarepta’s chief
executive officer. “We find this therapeutic approach to be of
particular interest as it has the potential to treat all patients
suffering from the disease regardless their mutation.”
The experimental program explores the potential surrogate gene
therapy approach to Duchenne muscular dystrophy. As a “surrogate
gene therapy approach”, the gene therapy looks to induce genes that
make proteins that can perform a similar function as dystrophin,
with the goal of producing a muscle cell that can function normally
even when dystrophin is absent. It has the potential to be used
broadly in several muscular dystrophies.
“We are pleased to have this opportunity to help advance our
Galgt2 gene therapy program,” said Dr. Kevin Flanigan, the
Principal Investigator leading the clinical trial. “Our goal is to
have this program in the clinic during 2017 and begin to evaluate a
therapy that has the potential to treat patients of all ages and
disease severity.”
About Sarepta Therapeutics
Sarepta Therapeutics is a 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 DMD
drug candidates. For more information, please visit us
at www.sarepta.com.
About The Research Institute at Nationwide Children's
Hospital
Named to the Top 10 Honor Roll on U.S. News & World
Report’s 2016-17 list of “America’s Best Children’s
Hospitals,” Nationwide Children’s Hospital is one of America’s
largest not-for-profit freestanding pediatric healthcare systems
providing wellness, preventive, diagnostic, treatment and
rehabilitative care for infants, children and adolescents, as well
as adult patients with congenital disease. As home to the
Department of Pediatrics of The Ohio State University College of
Medicine, Nationwide Children’s faculty train the next generation
of pediatricians, scientists and pediatric specialists. The
Research Institute at Nationwide Children’s Hospital is one of the
Top 10 National Institutes of Health-funded free-standing pediatric
research facilities in the U.S., supporting basic, clinical,
translational and health services research at Nationwide
Children’s. The Research Institute encompasses three research
facilities totaling 525,000 square feet dedicated to research. More
information is available at NationwideChildrens.org/Research.
Forward-Looking Statements
This 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
exclusive license agreement Sarepta has entered into with
Nationwide Children’s Hospital for their Galgt2 gene therapy
program, the potential for this therapeutic approach to treat all
patients suffering from DMD regardless of their mutation, age and
disease severity, the expected clinical progress of the program in
2017, the potential for the gene therapy to be used in several
muscular dystrophies, the gene therapy’s mechanism of action and
potential efficacy and Sarepta taking a multi-front approach in the
battle against DMD.
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 exclusive license agreement and
related agreements 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
partnership between Sarepta and Nationwide Children’s hospital 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 agreements; and even if the agreements result 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 2015 Annual
Report on Form 10-K and most recent Quarterly Report on Form 10-Q
for the quarter ended September 30, 2016 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 Information
We routinely post information that may be important to investors
in the 'For Investors' section of our web site
at www.sarepta.com. We encourage investors and potential
investors to consult our website regularly for important
information about us.
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version on businesswire.com: http://www.businesswire.com/news/home/20170110005563/en/
Media and Investors:Sarepta Therapeutics, Inc.Ian Estepan,
617-274-4052iestepan@sarepta.comorW2O GroupBrian Reid,
212-257-6725breid@w2ogroup.com
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