发明名称 Amine Cationic Lipids and Uses Thereof
摘要 The present invention relates to lipid compounds and uses thereof. In particular, the compounds include a class of cationic lipids having an amine moiety, such as an amino-amine or an amino-amide moiety. The lipid compounds are useful for in vivo or in vitro delivery of one or more agents (e.g., a polyanionic payload or an antisense payload, such as an RNAi agent).
申请公布号 US2014371293(A1) 申请公布日期 2014.12.18
申请号 US201414255637 申请日期 2014.04.17
申请人 Dicerna Pharmaceuticals, Inc. 发明人 Brown Bob Dale;Basu Sujit Kumar;Schwartz David A.;Fraser Allister
分类号 A61K47/18;A61K31/713;C12N15/113;A61K47/22 主分类号 A61K47/18
代理机构 代理人
主权项 1. A compound having the formula:or a pharmaceutically acceptable salt thereof, wherein each R1 and R2 is, independently, optionally substituted C11-24 alkyl, optionally substituted C11-24 alkenyl, optionally substituted C11-24 alkynyl, optionally substituted C11-24 heteroalkyl, optionally substituted C11-24 heteroalkenyl, or optionally substituted C11-24 heteroalkynyl, wherein said R1 and R2 is not substituted with an oxo on the carbon adjacent to >CHNR3R4; R3 is H or optionally substituted C1-6 alkyl; and R4 is unsubstituted C1-6 alkyl that is substituted with —NR4aR4b, substituted C1-6 alkyl that is further substituted with —NR4aR4b, or optionally substituted C3-7 heterocyclyl, wherein each R4a and R4b is, independently, H, C(═NH)NH2, or optionally substituted C1-6 alkyl, or wherein R4a and R4b combine together to form optionally substituted C3-7 heterocyclyl; or R3 and R4 combine together to form an optionally substituted C3-7 heterocyclyl, or R3 and R4 do not combine together and form optionally substituted imidazolyl or optionally substituted benzimidazolyl or optionally substituted succinimidyl; wherein one, and only one, primary amine can be present on either R3 or R4 or no primary amine is present on either R3 or R4; and wherein neither R3 nor R4 is an optionally substituted amide; and wherein when R1 or R2 is saturated C11 alkyl or saturated C15 alkyl, R3 is not H; wherein when R1 or R2 is saturated C16 alkyl or saturated C17 alkyl, R1 and R2 is not substituted with hydroxy; wherein when R1 or R2 is saturated C17 alkyl, R3 or R4 is not substituted with hydroxy; and wherein when R1 or R2 is saturated C18 alkyl, R4 is not substituted with optionally substituted imidazolyl.
地址 Watertown MA US