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LIPIDE CATIONIQUE IONISABLE POUR L'ADMINISTRATION D'ARN
专利权人:
ARCTURUS THERAPEUTICS; INC.
发明人:
PAYNE, JOSEPH E.,CHIVUKULA, PADMANABH
申请号:
CA2968060
公开号:
CA2968060A1
申请日:
2015.05.11
申请国别(地区):
CA
年份:
2016
代理人:
摘要:
What are described are compounds of formulas ?, HI, IV, and V. The compound of formula II consists of a compound in which R1 and R2 are both a linear alkyl consisting of 1 to 12 carbons, an alkenyl or alkynyl consisting of 2 to 12 carbons; L1 and L2 both consist of a linear alkylene or alkenylene consisting of 5 to 18 carbons, or forming a heterocycle with N; X is S; L3 consists of a bond or a linear alkylene consisting of 1 to 6 carbons, or farming a heterocycle with N; R3 consists of a linear or branched alkylene consisting of 1 to 6 carbons; and R4 and R5 are the same or different, each consisting of hydrogen or a linear or branched alkyl consisting of 1 to 6 carbons. The compound of formulas HI and IV consists of a compound in which R1 consists of a branched alkyl with 12 to 20 carbons; R2 consists of a linear alkyl with 5 to 10 carbons or a branched alkyl with 12 to 20 carbons; L1 and L2 each consists of a bond or a linear alkyl having 1 to 3 carbon atoms; X consists of S or O; L3 consists of a bond or a lower alkyl; R3 consists of a lower alkyl; and R4 and R5 are the same or different, each consisting of a lower alkyl. The compound of formula V consists of a compound in which R1 consists of a linear or branched alkyl consisting of 1-18 carbons, an alkenyl or alkynyl consisting of 2 to 12 carbons, or a cholesteryl; R2 consists of a 606338680v1 linear or branched alkyl or an alkenyl consisting of 1 to 18 carbons; L1 consists of a linear alkyl consisting of 5 to 9 carbons or, when R1 consists of a cholesteryl then L1 consists of a linear alkylene or alkenyl consisting of 3 to 4 carbons; X1 consists of -0-(CO) or -(CO)-0; X2 consists of S or O; La consists of a bond or a linear alkylene of 1 to 6 carbons; R3 consists of a linear or branched alkylene with 1 to 6 carbons; and R4 and R5 are the same or different, each consisting of a linear or branched alkyl of 1 to 6 carbons. The compound of formulas II, III, IV, and V may comprise a pharmaceutically acceptable salt
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