US 11,690,922 B2 · Lipid compound as well as lipid carrier, nucleic acid lipid nanoparticle composition and pharmaceutical preparation containing same

Abstract

The present invention belongs to the technical field of gene therapy, and particularly relates to a series of lipid compounds as well as a lipid carrier, nucleic acid lipid nanoparticle composition and pharmaceutical preparation containing the same. A compound having a structure of a formula (I) provided by the present invention can be used for preparing a lipid carrier together with other lipid compounds, and exhibits pH response, and the entrapment efficiency to a nucleic acid drug is high, which greatly improves in-vivo delivery efficiency of the nucleic acid drug; and furthermore, a lipid compound with a specific structure can be chosen as a lipid carrier based on an organ in which the nucleic acid drug needs to be enriched, having a good market application prospect.

Claims18

Dependent claims

A compound of a formula (I-2), or a pharmaceutically acceptable salt, stereoisomer, or chelate thereof,

wherein
A1, A2, A3, A4 and A5 are each independently one or more of —O(C═O)—, —(C═O)O—, —O—, —S—S—, —NRaC(═O), or —C(═O)NRa—;
B1, B2, B3 and B4 are each independently C1-8 alkylene;
B5 and B6 are each independently C1-8 alkylene;
R1 and R2 are each independently C4-8 branched alkyl;
R3 is hydrogen, hydroxyl, hydroxyl-substituted alkyl or 5- to 7-membered heterocyclyl;
wherein the 5- to 7-membered heterocyclic group is optionally substituted by C1-4 alkyl, and contains 1 to 4 ring-forming heteroatoms, and the heteroatoms are each independently N, O or S;
Ra is hydrogen or C1-8 alkyl;
and
b and c are each independently 0 or 1.

7 more elements

A compound selected from the group consisting of any one of the following compound, or a pharmaceutically acceptable salt, stereoisomer, or chelate thereof:

Dependencies are inferred from the claim text.

Facts

Application

17/870,313Filed 21 Jul 2022

Priority

CN 202110821782.521 Jul 2021

Published

US 2023/0093138 A123 Mar 2023

Granted

4 Jul 2023

Term ends

Not estimatedThe source records don’t support an estimate.

Classification24 codes

A61K 48/005
Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy:…
C07D 233/60
With only hydrogen atoms or radicals containing only hydrogen and carbon atoms, attached to ring carbon atoms: with hydrocarbon radicals,…
A61K 9/127
Dispersions; Emulsions: synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
A61K 9/145
Intimate drug-carrier mixtures characterised by the carrier, e.g. ordered mixtures, adsorbates, solid solutions, eutectica, co-dried,…: with…

Lipid compound as well as lipid carrier, nucleic acid lipid nanoparticle composition and pharmaceutical preparation containing same

Life of this patent

Lipid compound as well as lipid carrier, nucleic acid lipid nanoparticle composition and pharmaceutical preparation containing same

Life of this patent

Abstract

Claims18