CureDuchenne and Sarepta Therapeutics host webinar to provide Duchenne 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 along with Debra Miller, CEO and Founder of CureDuchenne.

1. FOR IMMEDIATE RELEASE
Contact: Joanne Forster
CureDuchenne
joanne@cureduchenne.org
310.498.7585
Sarepta Therapeutics, Inc. and CureDuchenne to Host Webinar on Duchenne Muscular
Dystrophy Programs Utilizing Sarepta’s RNA-Targeted Technology Platforms
NEWPORT BEACH, Calif. (July 20, 2018) – 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 1pm PDT/4pm EDT.
Participants must register in advance at: https://tinyurl.com/y7h8pcex.
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.”

2. 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 www.sarepta.com.
About CureDuchenne
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 www.cureduchenne.org, and follow us
on Facebook, Twitter and YouTube.
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