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Sarepta's investigational gene therapy shows strength in limb-girdle muscular dystrophy Type 2E

Published on 07/10/19 at 09:59am

Sarepta Therapeutics has revealed positive new data for its investigational gene therapy SRP-9003 in the treatment of limb-girdle muscular dystrophy Type 2E (LGMD2E). The nine-month findings were drawn from just three participants aged between four and 13.

The results showed that, following treatment with a the therapy at a dose of 5x1013vg/kg, mean creatine kinase (CK) was significantly reduced compared to baseline at 270 days, with all participants showing improvement from baseline over all four functional measures.

LGMD2E is a debilitating and progressive condition which causes wasting and weakness of the body’s muscles, beginning with those in the hips and shoulders and eventually spreading to the arms and legs. This leads to loss of the ability to walk independently by as early as a patient’s teen years, often ending in death before the age of 30, with no cure currently available.

The condition is caused by a lack of native beta-sarcoglycan protein, and SRP-9003 works by transducing skeletal and cardiac muscle with a gene that codes for the full-length version of the protein.

“We have now observed consistent functional improvements, in addition to high levels of expression of the missing protein of interest and strong results in related biomarkers, in both of our first cohorts for Duchenne muscular dystrophy (SRP-9001) and LGMD2E (SRP-9003). We intend to test one higher dose of SRP-9003 in LGMD2E participants, select our clinical dose and then advance our SRP-9003 programme, along with our other five LGMD programmes, as rapidly as possible,” commented Doug Ingram, Sarepta’s President and Chief Executive Officer. “With the results of our first LGMD2E cohort, Sarepta continues to build its gene therapy engine, an enduring model created to design, develop and bring to the medical and patient community transformative therapies for those living with, and too often dying from, rare genetic disease.”

Matt Fellows

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