21 February 2023 - - The first patient has been dosed in Study SRP-9003-102, US-based Sarepta Therapeutics, Inc. (NASDAQ: SRPT), a specialist in precision genetic medicine for rare diseases, said.

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 US-only study enrolling ambulant patients aged 18 years or older and non-ambulant patients, ages 4-50 years, using clinical process SRP-9003 material.

SRP-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 or cardiac complications.

Limb-girdle muscular dystrophies are genetic diseases that cause progressive, debilitating weakness and wasting that begins in muscles around the hips and shoulders before progressing to muscles in the arms and legs.

Sarepta's six LGMD gene therapy programs in development include LGMD2E/R4, LGMD2D/R3, LGMD2C/R5, LGMD2B/R2, LGMD2L/R12 and LGMD2A/R1, which together represent more than 70 % of known LGMD cases.

Patients with LGMD2E begin showing neuromuscular symptoms such as difficulty running, jumping and climbing stairs before age 10.

The disease, which is an autosomal recessive subtype of LGMD, progresses to loss of ambulation in the teen years and often leads to early mortality. There is currently no treatment or cure for LGMD2E.