Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a developer of innovative
RNA-targeted therapeutics, today announced the formation of a Strategic
& Scientific Advisory Board (SSAB) with three key appointments: Louis
Kunkel, PhD, Beverly Davidson, PhD, and Jeffrey Chamberlain, PhD.
The SSAB will work closely with Sarepta’s management team to advance the
Company’s platform technology in Duchenne muscular dystrophy (DMD),
identify multiple new applications for Sarepta’s proprietary PMO
chemistry in the neuromuscular rare disease and infectious disease
areas, as well as provide insight on novel technologies to complement
the Company’s pipeline.
"We are thrilled with the addition of these experts to our team of
advisors," said Edward Kaye, M.D., Sarepta’s interim chief executive
officer and chief medical officer. "The background and experience our
SSAB members bring to Sarepta will be invaluable as we move our
follow-on exon skipping therapy product candidates forward into later
stage clinical development, but also as we work towards the goal of
discovering complementary therapies to further improve the lives of
patients with DMD and expanding our RNA technology for other genetic
neuromuscular diseases. As we begin to build our SSAB, I am proud to
have leaders the caliber of Drs. Kunkel, Davidson, and Chamberlain as
founding members and look forward to working with them.”
The initial appointments to Sarepta’s Strategic & Scientific Advisory
Board include:
Louis M. Kunkel, PhD, Past Chief of the Division of Genetics and
currently a member of the Division of Genetics and Genomics, Boston
Children’s Hospital. Professor of Pediatrics and Genetics, Harvard
Medical School; Co-Director of the Senator Paul D. Wellstone Muscular
Dystrophy Cooperative Research Center of Worchester MA; HHMI
Investigator, 1987-2010; and Member of the National Academy of Sciences
and the American Academy of Arts and Sciences. Dr. Kunkel is an
internationally recognized geneticist with decades of experience and
scientific success in the understanding of the basis for muscular
dystrophies. Dr. Kunkel is responsible for the identification of the
gene and encoded protein, dystrophin. He has received numerous awards
and honors for scientific leadership and achievement and currently
serves as the committee chair for the Muscular Dystrophy Association
(MDA), a committee he has been a member of for over 15 years.
Beverly Davidson, PhD, Director of the Raymond G Perelman Center
for Cellular and Molecular Therapeutics and Arthur V. Meigs Chair in
Pediatrics, Children’s Hospital of Philadelphia; Professor in the
Department of Pathology and Laboratory Medicine, Perelman School of
Medicine at the University of Pennsylvania. Dr. Davidson’s research
focuses on the pathogenesis and therapy of hereditary neurogenetic
diseases and the role of noncoding RNAs in neural development. She is an
internationally known expert in the study of inherited neurological
diseases and the development of molecular therapies. Dr. Davidson has
been named a fellow by the American Association for the Advancement of
Science, received the NIH Mathilde Solowey Award, was elected to the
Advisory Council for the American Society of Gene and Cell Therapy and
NINDS council, and was past chair of the Medical Sciences Section for
the American Association for the Advancement of Science.
Jeffrey Chamberlain, PhD, Professor in the Departments of
Neurology, Medicine, and Biochemistry; McCaw Endowed Chair in Muscular
Dystrophy at the University of Washington; Director of the Senator Paul
D. Wellstone Muscular Dystrophy Cooperative Research Center of Seattle.
Dr. Chamberlain’s research is primarily focused on two major goals: to
develop a better understanding of the molecular basis of the
pathophysiology of muscle disorders, and to develop gene and cell
therapies that will correct and treat the muscular dystrophies. Dr.
Chamberlain is a close collaborator with a number of prominent
institutions, including Children’s Hospital of Seattle, Fred Hutchinson
Research Center, University of Rochester, and Harborview Medical Center.
Dr. Chamberlain also serves on the advisory boards for CureDuchenne,
Akashi Therapeutics, and Solid Ventures.
About Sarepta Therapeutics
Sarepta Therapeutics is a biopharmaceutical company focused on the
discovery and development of unique RNA-targeted therapeutics for the
treatment of rare, infectious and other diseases. The Company is
primarily focused on rapidly advancing the development of its
potentially disease-modifying DMD drug candidates, including its lead
DMD product candidate, eteplirsen, designed to skip exon 51. Sarepta is
also developing therapeutics for the treatment of infectious diseases
such as drug-resistant bacteria and other rare human diseases. For more
information, please visit us at www.sarepta.com.
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,” “may,”
“intends,” “prepares,” “looks,” “potential,” “possible” and similar
expressions are intended to identify forward-looking statements. These
forward-looking statements include statements relating to the ability of
the SSAB to advance Sarepta’s platform technology, identify new
applications of Sarepta’s proprietary PMO chemistry and provide insight
on novel technologies; and the value of SSAB member insights to and
Sarepta’s plans to move follow-on exons forward in clinical development
and to discover complementary therapies to further improve the lives of
DMD patients and expanding our RNA technology for other diseases.
These forward-looking statements involve risks and uncertainties,
many of which are beyond Sarepta’s control. Actual results could
materially differ from those stated or implied by these forward-looking
statements as a result of such risks and uncertainties. Known risk
factors include the following: we may not be able to capitalize on our
experience or that of the SSAB to advance candidates; we may not be able
to develop, advance or complete research or clinical development for our
pipeline of product candidates and the results of our ongoing research
and development efforts may not be positive, demonstrate safety or
efficacy and may not support the advancement of candidates; our other
product candidates and/or Sarepta’s anti-sense based technology platform
may not be successful and we may not discover or be able to obtain
complimentary therapies; there may be delays in our projected regulatory
timelines relating to conducting research and initiating new clinical
trials for our product candidates, or making a product commercially
available for various reasons including possible limitations of Company
resources and regulatory, court or agency decisions, such as decisions
by the United States Patent and Trademark Office with respect to patents
that cover our product candidates; manufacturing of candidates may not
be successful and any or all of the Company’s drug candidates may fail
in research or development or may not receive required regulatory
approvals for commercialization; we may need and may not be able to
obtain additional funds to conduct our planned research, development or
commercialization efforts and execute our business plans; and those
risks identified under the heading “Risk Factors” in Sarepta’s most
recent Annual Report on Form 10-K or Quarterly Report on Form 10-Q filed
with the Securities and Exchange Commission (SEC) as well as other SEC
filings made by the Company which you are encouraged to review.
Any of the foregoing risks could materially and adversely affect the
Company’s business, results of operations and the trading price of
Sarepta’s common stock. You should not place undue reliance on
forward-looking statements. Sarepta does not undertake any obligation to
publicly update its forward-looking statements based on events or
circumstances after the date hereof, except to the extent required by
applicable law or SEC rules.
Internet Posting of Information
We 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.
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