Muthiah Manoharan,Kallanthottathil G. Rajeev,Muthusamy Jayaraman,David Butler,Jayaprakash K. Nair,Martin Maier,Laxman Eltepu
申请号：
US15603069
公开号：
US20180043009A1
申请日:
2017.05.23
申请国别(地区):
US
年份:
2018
代理人:
摘要：
The present invention provides lipids that are advantageously used in lipid particles for the in vivo delivery of therapeutic agents to cells. In particular, the invention provides lipids having the following structurewherein R1 and R2 are each independently for each occurrence optionally substituted C10-C30alkyl, optionally substituted C10-C30alkenyl, optionally substituted C10-C30alkynyl, optionally substituted C10-C30acyl, or -linker-ligand； R3 is H, optionally substituted C1-C10alkyl, optionally substituted C2-C10alkenyl, optionally substituted C2-C10alkynyl, alkylhetrocycle, alkylphosphate, alkylphosphorothioate, alkylphosphorodithioate, alkylphosphonates, alkylamines, hydroxyalkyls, ω-aminoalkyls, ω-(substituted)aminoalkyls, ω-phosphoalkyls, ω-thiophosphoalkyls, optionally substituted polyethylene glycol (PEG, mw 100-40K), optionally substituted mPEG (mw 120-40K), heteroaryl, heterocycle, or linker-ligand； E is O, S, N(Q), C(O), N(Q)C(O), C(O)N(Q), (Q)N(CO)O, O(CO)N(Q), S(O), NS(O)2N(Q), S(O)2, N(Q)S(O)2, SS, O═N, aryl, heteroaryl, cyclic or heterocycle； and, Q is H, alkyl, ω-aminoalkyl, ω-(substituted)aminoalky, ω-phosphoalkyl or ω-thiophosphoalkyl.
