Sarepta Therapeutics Announces Initiation of VOYAGENE, a Clinical Study of SRP-9003 for the Treatment of Limb-Girdle Muscular Dystrophy Type 2E/R4

Sarepta Therapeutics, the leader in precision genetic medicine for rare diseases, today announced that the first patient has been dosed in Clinical Study, SRP-9003-102, (also known as VOYAGENE), for the treatment of limb-girdle muscular dystrophy Type 2E/R4 (LGMD2E). VOYAGENE is a U.S.-only study enrolling ambulant patients aged 18 years or older and non-ambulant patients, ages 4-50 years, using clinical process SRP-9003 material.

“Sarepta has previously disclosed positive expression and function data from our initial clinical study, Study SRP-9003-101, exploring multiple doses of SRP-9003 in a younger, ambulant population. The VOYAGENE study, which also utilizes clinical material, provides an opportunity to generate additional data in a broader population of patients while we finalize plans for a global, Phase 3 study using commercially representative process material that we intend to begin later this year,” said Louise Rodino-Klapac, Ph.D., executive vice president, chief scientific officer and head of research and development, Sarepta Therapeutics.

“Sarepta is applying lessons from across our gene therapy portfolio to help advance the development of our investigational LGMD programs as efficiently as possible. Current treatments for LGMD are limited to symptom management and we remain fully committed to working with urgency to bring forward new treatments that can slow the progression of this disease, improve mobility and enhance the quality of life for individuals living with LGMD2E and other LGMD subtypes.”