Shares of Sarepta Therapeutics SRPT closed 8.4% higher on Tuesday, and were up as much as 12% in earlier in the trading session after it announced positive data for its Duchenne Muscular Dystrophy (DMD) drug.
SRP-9001 is an investigational gene transfer therapy intended to deliver a micro-dystrophin-encoding gene to muscle tissue. DMD causes muscle degeneration and weakness and alters thedystrophin protein that helps keep muscle cells intact.
20 participants between the ages of 4 and 7 were treated in the study. All showed robust transduction, with average dystrophin level of 55.4% of normal. Overall, SRP-9001 demonstrated “[a] consistent safety profile.” Mizuho Securities analyst Difei Yang told clients in a note that these positive results are an important step for Sarepta to begin Phase 3 testing.
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