SYMMETRIC IONIZABLE CATIONIC LIPID FOR RNA DELIVERY

摘要

What are described are compounds of formulas II, III, and IV. 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 forming 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 III 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 formulas II, III and IV may comprise a pharmaceutically acceptable salt thereof.

主权项

1. A compound of formula IIwherein
R1 and R2 both consist of 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 is a bond or a linear alkylene consisting of 1 to 6 carbons, or forming a heterocycle with N, R3 is a linear or branched alkylene consisting of 1 to 6 carbons, and R4 and R5 are the same or different, each hydrogen or a linear or branched alkyl consisting of 1 to 6 carbons.