Press release
Sarepta Therapeutics Announces Initiation of VOYAGENE, a Clinical Study of SRP-9003 for the Treatment of Limb-Girdle Muscular Dystrophy Type 2E/R4
CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced
About this update from Sarepta Therapeutics, Inc.
[{"type":"text","content":" CAMBRIDGE, Mass.--(BUSINESS WIRE)--\nSarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that the first patient has been dosed in Study SRP-9003-102. Also known as VOYAGENE, Study 9003-102 is a phase 1 study of SRP-9003 (bidridistrogene xeboparvovec) 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.\n\n“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.”\n\nMore information on the VOYAGENE study is available at https://genesislgmd.com/study/voyagene.\n\nAbout SRP-9003 (bidridistrogene xeboparvovec)\n\nSRP-9003 (bidridistrogene xeboparvovec) is an investigational gene therapy that uses the AAVrh74 vector, which is designed to be systemically and robustly delivered to skeletal, diaphragm and cardiac muscle, making it an ideal candidate to treat peripheral neuromuscular diseases. SRP-9003 also uses the MHCK7 promoter, chosen for its ability to robustly express in the heart, which is critically important for patients with limb-girdle muscular dystrophy Type 2E (LGMD2E), also known as beta-sarcoglycanopathy and LGMDR4, many of whom die from pulmonary ...