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美國Sarepta Therapeutics   
公司是一家生物制藥公司，研究和開發(fā)以抗轉(zhuǎn)錄療法和癌癥免疫療法技術(shù)為基礎(chǔ)的治療性產(chǎn)品.該公司成立于1980年，總部在美國華盛頓， 

 is focused on developing first-in-class RNA-based therapeutics to improve and save the lives of people affected by serious and life-threatening rare and infectious diseases. The Company's diverse pipeline includes its lead program eteplirsen, for Duchenne muscular dystrophy, as well as potential treatments for some of the world's most lethal infectious diseases. Sarepta aims to build a leading, independent biotech company dedicated to translating its RNA-based science into transformational therapeutics for patients who face significant unmet medical needs.