US 2025/0188013 A1 · LIPID COMPOUND AND USE THEREOF

Abstract

The present application provides a novel lipid compound, a lipid nanoparticle comprising the same, and use thereof in targeted delivery of a drug. Specifically, the present application provides a compound of Formula I or a pharmaceutically acceptable salt, a prodrug, or a stereoisomer thereof. Furthermore, the present application provides a lipid nanoparticle comprising the compound of Formula I or a pharmaceutically acceptable salt, a prodrug, or a stereoisomer thereof; still further, the lipid nanoparticle may further comprise a cationic lipid; yet still further, the lipid nanoparticle may further comprise a helper lipid. A composition comprising the above compound or a pharmaceutically acceptable salt, a prodrug, or a stereoisomer thereof, or the lipid nanoparticle can achieve the organ-targeted delivery of a therapeutic/prophylactic agent.

A lipid compound of formula I or a pharmaceutically acceptable salt, a prodrug, a stereoisomer, or a deuteride thereof,

wherein L1 is absent, —C—C—, —C═C—, —C≡C—, —O(C═O)—, —(C═O)O—, —C(═O)—, —O(C═O)O—, —O—, —S(O)x—, —S—S—, —C(═O)S—, —SC(═O)—, —NRaC(═O)—, —C(═O)NRa—, —NRaC(═O)NRa—, —OC(═O)NRa—, —NRaC(═O)O—, —NRa(CH2)x—, or —(CH2)xNRa—;
Q1 is absent or C1-C8 linear or branched hydrocarbyl;
L2 is absent, —C—C—, —C═C—, —C≡C—, —O(C═O)—, —(C═O)O—, —C(═O)—, —O(C═O)O—, —O—, —S(O)x—, —S—S—, —C(═O)S—, —SC(═O)—, —NRaC(═O)—, —C(═O)NRa—, —NRaC(═O)NRa—, —OC(═O)NRa—, or —NRaC(═O)O—;
Q2 is absent or C1-C8 linear or branched hydrocarbyl;
L3 is absent, —C—C—, —C═C—, —C≡C—, —O(C═O)—, —(C═O)O—, —C(═O)—, —O(C═O)O—, —O—, —S(O)x—, —S—S—, —C(═O)S—, —SC(═O)—, —NRaC(═O)—, —C(═O)NRa—, —NRaC(═O)NRa—, —OC(═O)NRa—, or —NRaC(═O)O—;
Q3 is absent or C1-C8 linear or branched hydrocarbyl;
Ra is H, deuterium, C1-C8 linear or branched hydrocarbyl, or C1-C8 linear or branched hydrocarbyl substituted with carboxyl;
X is C or N;
R is
Z is C, O, NRb, or S;
Rb is H, deuterium, or C1-C8 linear or branched hydrocarbyl;
one of Y1 and Y2 is absent, —C—C—, —C═C—, —C≡C—, —O(C═O)—, —C(OH)—, —(C═O)O—, —C(═O)—, —O(C═O)O—, —O—, —S(O)x—, —S—S—, —C(═O)S—, —SC(═O)—, —NRaC(═O)—, —C(═O)NRa—, —NRaC(═O)NRa—, —OC(═O)NRa—, or —NRaC(═O)O—, and the other one of Y1 and Y2 is absent, —C—C—, —C═C—, —C≡C—, —O(C═O)—, —C(OH)—, —(C═O)O—, —C(═O)—, —O(C═O)O—, —O—, —S(O)x—, —S—S—, —C(═O)S—, —SC(═O)—, —NRaC(═O)—, —C(═O)NRa—, —NRaC(═O)NRa—, —OC(═O)NRa—, or —NRaC(═O)O—;
G1 and G2 are each independently absent or substituted C1-C12 linear or branched hydrocarbyl;
one of Y3 and Y4 is absent, —C—C—, —C═C—, —C≡C—, —O(C═O)—, —C(OH)—, —(C═O)O—, —C(═O)—, —O(C═O)O—, —O—, —S(O)x—, —S—S—, —C(═O)S—, —SC(═O)—, —NRaC(═O)—, —C(═O)NRa—, —NRaC(═O)NRa—, —OC(═O)NRa—, or —NRaC(═O)O—, and the other one of Y3 and Y4 is absent, —C—C—, —C═C—, —C≡C—, —O(C═O)—, —C(OH)—, —(C═O)O—, —C(═O)—, —O(C═O)O—, —O—, —S(O)x—, —S—S—, —C(═O)S—, —SC(═O)—, —NRaC(═O)—, —C(═O)NRa—, —NRaC(═O)NRa—, —OC(═O)NRa—, or —NRaC(═O)O—;
G3 and G4 are each independently absent or substituted C1-C12 linear or branched hydrocarbyl;
one of Y5 and Y6 is absent, —C—C—, —C═C—, —C≡C—, —O(C═O)—, —C(OH)—, —(C═O)O—, —C(═O)—, —O(C═O)O—, —O—, —S(O)x—, —S—S—, —C(═O)S—, —SC(═O)—, —NRaC(═O)—, —C(═O)NRa—, —NRaC(═O)NRa—, —OC(═O)NRa—, or —NRaC(═O)O—, and the other one of Y5 and Y6 is absent, —C—C—, —C═C—, —C≡C—, —O(C═O)—, —C(OH)—, —(C═O)O—, —C(═O)—, —O(C═O)O—, —O—, —S(O)x—, —S—S—, —C(═O)S—, —SC(═O)—, —NRaC(═O)—, —C(═O)NRa—, —NRaC(═O)NRa—, —OC(═O)NRa—, or —NRaC(═O)O—;
Ra is H, deuterium, C1-C8 linear or branched hydrocarbyl, or C1-C8 linear or branched hydrocarbyl substituted with carboxyl;
G5 and G6 are each independently absent or substituted C1-C12 linear or branched hydrocarbyl;
R1 and R2 are each independently substituted C1-C24 linear or branched hydrocarbyl;
x is 0, 1, or 2.

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Lipid compound and use thereof

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Lipid compound and use thereof

Life of this patent

Abstract

Claims33