Sarepta posts early, rosy gene therapy data for limb girdle muscular dystrophy — fueling $165M buyout of partner
Sarepta Therapeutics $SRPT has revealed a promising, early snapshot of results for its second gene therapy program in patients with a progressive muscle degenerative disorder — months after reporting encouraging data for its Duchenne gene therapy, as the company fortifies its status as a bonafide gene therapy player. And the results inspired a $165 million buyout.
On Wednesday, Sarepta divulged data from three patients in the first cohort of an open-label Phase I/II study testing the use of an experimental gene therapy — MYO-101 — in limb girdle muscular dystrophy (LGMD), a group of rare progressive genetic disorders characterized by wasting and weakness of the voluntary muscles of the hip and shoulder areas (limb-girdle area) that have no approved treatments.
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