EMERGENE Phase 3 Study Begins for Sarepta's LGMD2E/R4 Gene Therapy

Sarepta Therapeutics, a frontrunner in precision genetic medicine for rare diseases, has commenced participant screening for the Phase 3 clinical trial known as EMERGENE, or Study SRP-9003-301. This study is dedicated to evaluating the efficacy of SRP-9003 (bidridistrogene xeboparvovec), an experimental gene therapy, in treating limb-girdle muscular dystrophy Type 2E (LGMD2E/R4), also known as beta sarcoglycanopathy. The condition is a rare form of LGMD that currently lacks treatment options beyond symptom management.

The EMERGENE study is set to include 15 participants, both ambulatory and non-ambulatory, who are aged four and above. The study will utilize a commercially representative process of SRP-9003 material. Louise Rodino-Klapac, Ph.D., Sarepta's executive vice president and head of research and development, expressed enthusiasm for the progress of SRP-9003. She highlighted early clinical results showing significant protein expression and functional benefits, such as slowing disease progression, improving mobility, and enhancing the quality of life for those affected by LGMD2E.

SRP-9003 is an investigational gene therapy designed to be delivered systemically to skeletal, diaphragm, and cardiac muscle using the AAVrh74 vector. It aims to provide a full-length beta-sarcoglycan transgene, which is crucial for patients with LGMD2E, many of whom face pulmonary or cardiac complications.

The Phase 3 study will assess the expression of beta-sarcoglycan 60 days post-treatment, with additional functional measures and safety evaluations continuing through the 60th month. Limb-girdle muscular dystrophies are genetic disorders characterized by progressive muscle weakness and wasting, starting in the muscles around the hips and shoulders and eventually affecting the limbs.

Sarepta Therapeutics is focused on developing precision genetic medicine for rare diseases, with a robust pipeline of over 40 programs. The company holds leadership positions in Duchenne muscular dystrophy (DMD) and limb-girdle muscular dystrophies (LGMDs) and is driven by a multi-platform approach that includes gene therapy, RNA, and gene editing technologies.

LGMD2E patients typically exhibit symptoms before the age of 10, with the disease progressing to loss of ambulation in the teenage years and often resulting in early mortality. Currently, there are no disease-modifying treatments available for LGMD2E. Sarepta's commitment to advancing SRP-9003 and its other LGMD programs represents a significant step towards addressing the unmet needs of patients with these rare conditions.