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Sarepta Therapeutics, Inc. and CureDuchenne to Host Webinar on Duchenne Muscular Dystrophy Programs Utilizing Sarepta's RNA-Targeted Technology Platforms


Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a commercial-stage
biopharmaceutical company focused on the discovery and development of
precision genetic medicine to treat rare neuromuscular diseases, and
non-profit CureDuchenne announced today they have partnered to host a
free webinar to provide Duchenne muscular dystrophy patients, caregivers
and advocates a comprehensive review of Sarepta's RNA-targeted
technology platforms (PMO and PPMO). Gilmore O'Neill, M.B., M.M.Sc.,
Chief Medical Officer of Sarepta, will present on behalf of the Company.

The hour-long webinar will take place on Wednesday, July 25, 2018, at 1
p.m. PDT/4 p.m. EDT. Participants must register in advance at:

Dr. O'Neill will highlight PPMO, Sarepta's next-generation chemistry
platform designed around a proprietary cell-penetrating peptide
conjugated to the PMO backbone, with the goal of increasing tissue
penetration, increasing exon skipping and significantly increasing
dystrophin production. If successful, the PPMO offers the potential for
improved efficacy and less frequent dosing for patients.

In addition, Dr. O'Neill will discuss Sarepta's enrolling clinical
trial, 5051-101, the first-in-human Phase I PPMO trial and its open
label extension trial, 5051-102.

SRP-5051 uses Sarepta's PPMO chemistry and exon-skipping technology to
skip exon 51 of the dystrophin gene. SRP-5051 is designed to bind to
exon 51 of dystrophin pre-mRNA, resulting in exclusion of this exon
during mRNA processing in patients with genetic mutations that are
amenable to exon 51 skipping. Exon skipping is intended to allow for
production of an internally truncated dystrophin protein.

"Sarepta is dedicated to rapidly advancing precision genetic medicines
that address the underlying cause of Duchenne," said Dr. O'Neill.
"Toward that end, we have 16 programs in various stages of pre-clinical
and clinical development. I am honored and very much looking forward to
the opportunity to address the Duchenne community about the scientific
underpinnings of our RNA-targeted technology platforms, and the status
and direction of our promising clinical trials."

About Sarepta Therapeutics

Sarepta Therapeutics is a commercial-stage biopharmaceutical company
focused on the discovery and development of a precision genetic medicine
to treat 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

About CureDuchenne

is the nation's leading nonprofit organization dedicated to finding a
cure for Duchenne, the most common and most lethal form of muscular
dystrophy. As the leading genetic killer of young boys, Duchenne affects
more than 300,000 patients worldwide, most of them boys and young men.
CureDuchenne has garnered international attention for its efforts to
raise funds and awareness for Duchenne through venture philanthropy and
to improve care through CureDuchenne Cares regional programs. For more
information on how to help raise awareness and funds needed for
research, please visit,
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