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    • 1. 发明申请
    • COMPLEXES FOR THE DELIVERY OF BIOLOGICALLY-ACTIVE MATERIAL TO CELLS
    • 用于将生物活性材料递送至细胞的复合物
    • WO2003094974A1
    • 2003-11-20
    • PCT/GB2003/001985
    • 2003-05-08
    • UNIVERSITY COLLEGE LONDONHURLEY, Christopher, AntonyHAILES, Helen, ClaireTABOR, Alethea, BerniceHART, Stephen, Lewis
    • HURLEY, Christopher, AntonyHAILES, Helen, ClaireTABOR, Alethea, BerniceHART, Stephen, Lewis
    • A61K47/48
    • A61K47/543
    • A complex suitable for delivery of a biologically-active material to a cell, which complex comprises: (i) a lipid of general formula (I) or (II): wherein: -X 1 and X 2 are the same or different and are selected from -O-CH 2 -, and -O-C(O)-; -R 1 and R 2 are the same or different and are straight or branched, saturated or unsaturated C 7 to C 24 hydrocarbyl groups which are unsubstituted or substituted by one or more substituents selected from hydroxy, halogen and OR', wherein R' is a C 1 to C 6 hydrocarbyl group; -Each R 3 and each R 4 is the same or different and is a straight or branched, saturated or unsaturated C 1 to C 10 hydrocarbyl group which is unsubstituted or substituted by one or more substituents selected from hydroxy, halogen, -OR', -C(O)OH, -CN, -NR'R", and -C(O)R" wherein R' and R" are the same or different and are C 1 to C 6 hydrocarbyl; (Formula II) Wherein: X 1 and X 2 are the same or different and are as defined above; R 1 and R 2 are the same or different and are as defined above; R 5 is -N ⊕ (R 3 ) 2 -R 6 wherein each R 3 is the same or different and is as defined above and R 6 is either: (a) (Formula A) wherein each Y is the same or different and is -N ⊕ (R 4 ) 2 -, wherein R 4 is as defined above, each A is the same or different and is a C 1 to C 20 alkylene group which is unsubstituted or substituted by one or more substituents selected from hydroxy, halogen, -OR', -C(O)OH, -CN, -NR'R", and -C(O)R" wherein R' and R" are the same or different and are C 1 to C 6 hydrocarbyl, n is from 1 to 10, and R 4 is as defined above; or (b)-(Formula (B) wherein each B is the same or different and is a C 1 to C 10 alkylene group which is unsubstituted or substituted by one or more substituents selected from hydroxy, halogen, -OR', -C(O)OH, -CN, -NR'R" and -C(O)R" wherein R' and R" are the same or different and are C 1 to C 6 hydrocarbyl, m is from 1 to 10, and Q is selected from N ⊕ (R 3 ) 3 , OH, OR', OC(O)R' and halogen, wherein R 3 and R' are as defined above; and (ii) A biologically-active material. Such complexes may be used to deliver biologically active material to a cell, for example in gene therapy and vaccination.
    • 一种适于将生物活性物质递送至细胞的复合物,该复合物包括:(i)通式(I)或(II)的脂质:其中:-X 1和X 2相同 或不同的并且选自-O-CH 2 - 和-OC(O) - ; -R 1和R 2相同或不同,是未取代或被一个或多个选自羟基,卤素和OR'的取代基取代的直链或支链,饱和或不饱和的C 7至C 24烃基,其中R '是C1至C6烃基; - 每个R 3和每个R 4相同或不同,并且是未取代的或被一个或多个选自羟基,卤素-OR的取代基取代的直链或支链,饱和或不饱和的C1至C10烃基 ',-C(O)OH,-CN,-NR'R“和-C(O)R”,其中R'和R“相同或不同,为C 1至C 6烃基;(式II)其中: X 1和X 2相同或不同,如上所定义; R 1和R 2相同或不同,如上所定义; R 5为-N <⊕> (R 3)2 -R 6其中每个R 3相同或不同,如上所定义,R 6是:(a)(式A)其中每个Y相同或 不同,为-N≡(R 4)2-,其中R 4如上所定义,各A相同或不同,为未取代的或被一个或多个取代基取代的C 1〜C 20亚烷基 选自羟基,卤素,-OR', - C(O)OH,-CN,-NR'R“和-C(O)R”中的更多取代基,其中R'和R“相同或不同,为C1 至C6氢化 芳基,n为1至10,R 4如上定义; 或(b) - (式(B))其中每个B相同或不同,并且是未取代的或被一个或多个选自羟基,卤素,-OR',-C(O) )OH,-CN,-NR'R“和-C(O)R”,其中R'和R“相同或不同,为C1至C6烃基,m为1至10,Q选自N (ⅲ)3,OH,OR',OC(O)R'和卤素,其中R 3和R'如上所定义;和(ⅱ)生物活性物质, 可用于将生物活性物质递送至细胞,例如在基因治疗和疫苗接种中。