作者: Alnylam引领了siRNA赛道的高速发展,2024年还将迎来多个里程碑事件,ALN-TTRsc将启动三期临床,ALN-APP将启动二期临床,此外与赛诺菲合作的血友病siRNA疗法也将递交上市申请。 遥遥领先! 引用: 2024-06-24 20:45 ▎Armstrong 2024年6月24日,Alnylam公布Vutrisiran治疗ATTR-CM三期临床HELIOS-B最新数据,总体人群和...
Phase I First-in-Humans Trial of ALN-TTRsc, a Novel RNA Interference Therapeutic for the Treatment of Familial Amyloidotic Cardiomyopathy (FAC)[J] . Tracy Zimmermann,Verena Karsten,Jamie Harrop,Amy Chan,Joseph Chiesa,Gary Peters,Rick Falzone,Jeffrey Cehelsky,Saraswathy Nochur,Akshay Vaishnaw,Jared...
ALN-TTRsc administration resulted in an approximately 80% reduction of TTR at doses as low as 2.5 mg/kg. In single- and multi-dose pre-clinical safety studies in rodents and non-human primates, ALN-TTRsc was found to be generally
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