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RNA IONIZABLE CATIONIC LIPID FOR RNA DELIVERY
专利权人:
아크투루스 쎄라퓨틱스, 인크.
发明人:
페인 조셉 이.,치부쿨라 파드마나브
申请号:
KR1020177016742
公开号:
KR1020170095241A
申请日:
2015.05.11
申请国别(地区):
KR
年份:
2017
代理人:
摘要:
Described are compounds of formula II, formula III, formula IV, and formula V. The compounds of formula II are those wherein R 1 and R 2 are both linear alkyl of 1 to 12 carbons, alkenyl or alkynyl of 2 to 12 carbons; Both of L <; 1>; and L <; 2 >; are linear alkylene or alkenylene consisting of 5 to 18 carbons, or together with N form a heterocycle; X is S; L 3 is a bond or linear alkylene of 1 to 6 carbons, or forms a heterocycle together with N; R 3 is a linear or branched alkylene of 1 to 6 carbons; R 4 and R 5 are the same or different and each is a hydrogen or a compound consisting of linear or branched alkyl of 1 to 6 carbons. Compounds of formula III and formula IV are characterized in that R <; 1 >; is a branched alkyl having 12 to 20 carbons; R <; 2 >; is linear alkyl having 5 to 10 carbons or branched alkyl having 12 to 20 carbons; Each of L 1 and L 2 is a bond or a linear alkyl having 1 to 3 carbon atoms; X is S or O; L <; 3 >; is a bond or lower alkyl; R <; 3 >; is lower alkyl; R 4 and R 5 are the same or different and each is composed of a compound consisting of lower alkyl. The compounds of formula V are those wherein R <; 1 >; is linear or branched alkyl of from 1 to 18 carbons, alkenyl or alkynyl of from 2 to 12 carbons, or cholesteryl; R <; 2>; is 606338680 v1 linear or branched alkyl or alkenyl having 1 to 18 carbons; L 1 is linear alkyl of 5 to 9 carbons, or, when R 1 is cholesteryl, L 1 is linear alkylene or alkenyl of 3 to 4 carbons; X 1 is -O- (CO) - or - (CO) -O-; X <; 2 >; is S or O; La is a bond or linear alkylene of 1 to 6 carbons; R 3 is a linear or branched alkylene having 1 to 6 carbons; R 4 and R 5 are the same or different and each is composed of a compound consisting of linear or branched alkyl of 1 to 6 carbons. The compounds of formula (II), (III), (IV) and (V) may include a pharmaceutically acceptable salt thereof.기술된 것은 화학식 II, 화학식 III, 화학식 IV, 및 화학식 V의 화합물이다. 화학식 II의 화합물은 R1 및 R2 둘 다가 1 내지
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