Sarep­ta goes back to the gene ther­a­py well at Na­tion­wide Chil­dren’s for its lat­est mus­cle-wast­ing pro­gram

Sarep­ta $SRPT is adding a pre­clin­i­cal gene ther­a­py pro­gram to the pipeline, pick­ing it up from their close con­tacts at Na­tion­wide Chil­dren’s Hos­pi­tal. The treat­ment is cal­pain 3 (CAPN-3), for Limb-gir­dle mus­cu­lar dy­s­tro­phy (LGMD) type 2A, which trig­gers the dis­ease as­so­ci­at­ed with weak­ness in mus­cles in the hips and shoul­ders.

This is the biotech’s 6th LGMD ef­fort, which us­es an AAVrh74 vec­tor to tar­get skele­tal mus­cle. The work so far has been led by Zarife Sa­henk, di­rec­tor of Clin­i­cal and Ex­per­i­men­tal Neu­ro­mus­cu­lar Pathol­o­gy at the Re­search In­sti­tute at Na­tion­wide Chil­dren’s in Ohio.

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