Sarepta Therapeutics, Inc. 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).
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: 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.”
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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