Sarepta therapeutics' investigational gene therapy srp-9003 for the treatment of limb-girdle muscular dystrophy type 2e shows sustained expression and functional improvements 2 years after administration

-- protein expression in muscle was sustained for two years following treatment in the low dose cohort, with mean beta-sarcoglycan expression of 54% at 24 months, compared to 36% at day 60, as measured by western blot -- -- mean nsad score improvement of 5.7 points from baseline was sustained through 24 months in low-dose cohort, and mean nsad score improvement of 4.0 points from baseline at one year in high-dose cohort -- -- results in both cohorts continue to reinforce the safety and tolerability profile of srp-9003 -- cambridge, mass., march 18, 2021 (globe newswire) -- sarepta therapeutics, inc. (nasdaq:srpt), the leader in precision genetic medicine for rare diseases, today shared new results from the ongoing study of srp-9003 (raavrh74.mhck7.hsgcb), the company's investigational gene therapy for limb-girdle muscular dystrophy type 2e (lgmd2e). in the first look at expression data from biopsies taken two years after a single administration of srp-9003, results found sustained protein expression in muscle tissue. in functional outcomes assessments taken two years following treatment in cohort 1 (low-dose cohort) and one year after treatment in cohort 2 (high-dose cohort), patients continued to demonstrate stability in their nsad (north star assessment for dysferlinopathies) total score and improvements on timed function tests. results are being presented today at the 2021 muscular dystrophy association (mda) annual clinical and scientific conference.
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