申请人:Massachusetts Institute of Technology
公开号:US20140329884A1
公开(公告)日:2014-11-06
The present invention provides novel 1,3,5-triazinane-2,4,6-trione derivatives, such as compounds of any one of Formulae (I) and (II), and salts thereof, and methods of preparing the compounds. Also provided are compositions including a compound of the invention and an agent (e.g., an siRNA, mRNA, or plasmid DNA). The present invention also provides methods and kits using the compositions for delivering an agent to a subject (e.g., to the liver, spleen, or lung of the subject) or cell and for treating and/or preventing a range of diseases, such as genetic diseases, proliferative diseases, hematological diseases, neurological diseases, liver diseases, and lung diseases.
本发明提供了新型的1,3,5-三嗪烷-2,4,6-三酮衍生物,例如公式(I)和(II)中的任一化合物及其盐,并提供了制备这些化合物的方法。还提供了包括本发明化合物和一种药剂(例如siRNA、mRNA或质粒DNA)的组合物。本发明还提供了使用该组合物将药剂传递到受体(例如受体的肝脏、脾脏或肺)或细胞以及治疗和/或预防一系列疾病(例如遗传性疾病、增殖性疾病、血液疾病、神经系统疾病、肝脏疾病和肺部疾病)的方法和试剂盒。