1,3,5-triazinane-2,4,6-trione derivatives and uses thereof
    9.
    发明授权
    1,3,5-triazinane-2,4,6-trione derivatives and uses thereof 有权
    1,3,5-三氮杂环戊烷-2,4,6-三酮衍生物及其用途

    公开(公告)号:US09315472B2

    公开(公告)日:2016-04-19

    申请号:US14267530

    申请日:2014-05-01

    摘要: 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.

    摘要翻译: 本发明提供新的1,3,5-三氮杂环丁烷-2,4,6-三酮衍生物,如式(I)和(II)中任一项的化合物及其盐,以及制备该化合物的方法。 还提供了包含本发明化合物和试剂(例如siRNA,mRNA或质粒DNA)的组合物。 本发明还提供了使用组合物将试剂递送至受试者(例如,受试者的肝脏,脾脏或肺)或细胞的方法和试剂盒,并用于治疗和/或预防一系列疾病,例如遗传 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。