Immunohistochemistry for alpha-sarcoglycan in A) normal muscle, B) LGMD2D (adhalin deficiency). Credit: Jensflorian/ commons.wikimedia.org.

Sarepta Therapeutics has dosed the first participant in the Phase I VOYAGENE clinical trial of SRP-9003 (bidridistrogene xeboparvovec) to treat limb-girdle muscular dystrophy (LGMD) type 2E/R4 (LGMD2E).

The VOYAGENE study has been designed for evaluating SRP-9003, an investigational gene therapy, in patients with LGMD2E, which is caused by a non-working β-sarcoglycan gene.

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It will find out whether it is safe to add a new, functional version of this gene to a person’s body.

The US-only study is enrolling ambulant patients aged 18 years or above, as well as non-ambulant patients aged four to 50 years, using clinical process SRP-9003 material.

Researchers have developed a version of the gene that will be sent into the patient’s body. The trial will also find out the effect the gene may have on LGMD.

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SRP-9003 uses the AAVrh74 vector designed to be delivered to skeletal, diaphragm, and cardiac muscle for the treatment of peripheral neuromuscular diseases.

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It also leverages the MHCK7 promoter, which is selected for its ability to express in the heart.

MHCK7 promoter is said to be critically important for LGMD2E patients.

Sarepta Therapeutics executive vice-president, chief scientific officer and research and development head Louise Rodino-Klapac said: “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 utilises clinical material, provides an opportunity to generate additional data in a broader population of patients while we finalise plans for a global, Phase III study using commercially representative process material that we intend to begin later this year.

“Sarepta is applying lessons from across our gene therapy portfolio to help advance the development of our investigational LGMD programmes as efficiently as possible.”

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