Screening is initiated in EMERGENE, a phase III clinical study of SRP 9003 for the treatment of limb-girdle muscular dystrophy type 2E/R4

EMERGENE will enroll 15 participants (ambulatory and non-ambulatory), aged 4 and older, and uses commercially representative process SRP 9003 material.

“We are pleased to share our continued progress in advancing SRP 9003, our investigational gene therapy candidate for LGMD2E, a rare form of LGMD with no treatments beyond symptom management. Early results from the SRP 9003 clinical development program demonstrated significant protein expression at both 12-weeks and two years after treatment as well as functional benefits including slowing progression of this disease, improving mobility, and enhancing the quality of life for individuals living with LGMD2E,” said Louise Rodino-Klapac, Ph.D., executive vice president, chief scientific officer and head of research and development, Sarepta Therapeutics. “In addition to its importance for the LGMD2E community, EMERGENE will inform the clinical development of other programs for LGMD in Sarepta’s pipeline while serving as a pathfinder for viable regulatory pathways to support the development of gene therapies to treat ultra rare diseases.”

About Study SRP-9003-301 (EMERGENE): EMERGENE, Study 9003-301 is a Phase III, multinational, open-label study of SRP 9003 for the treatment of LGMD2E in 15 ambulatory and non-ambulatory participants, ages 4 and older. The EMERGENE design incorporates a six-month natural history lead-in. The primary endpoint is expression of beta-sarcoglycan 60 days after dosing. Other endpoints include functional measures through month 60 and safety.