美国Sarepta Therapeutics   
公司是一家生物制药公司，研究和开发以抗转录疗法和癌症免疫疗法技术为基础的治疗性产品.该公司成立于1980年，总部在美国华盛顿， 

 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.