NOVEL CATIONIC LIPID COMPOUND

WIPO Patent Application WO/2023/015904

A1

The present invention relates to a lipid compound, which can be used alone, or can be used in combination with other lipid components such as neutral lipids, charged lipids, steroids and/or analogues thereof, and/or polymer-conjugated lipids to form a lipid nanoparticle for delivering a therapeutic agent and/or a prophylactic agent. In some examples, the lipid nanoparticle is used for delivering nucleic acids, such as a messenger RNA and/or an antisense RNA. Further provided is a method for treating and/or preventing various diseases by means of using such lipid nanoparticles. In one embodiment, provided is a compound with a structure of formula (I) as shown below: or a salt, an isomer or a N-oxide thereof, wherein R1 is as defined herein. Also provided is a pharmaceutical composition comprising one or more compounds of structural formula (I) above and a therapeutic agent and/or a prophylactic agent. In some embodiments, the pharmaceutical composition also comprises one or more components selected from neutral lipids, charged lipids, steroids and polymer-conjugated lipids. Such compositions are useful for forming a lipid nanoparticle for delivering the therapeutic agent and/or the prophylactic agent. In other embodiments, provided in the present invention is a method for administering the therapeutic agent and/or the prophylactic agent to a subject in need thereof, wherein the method comprises preparing a pharmaceutical combination of the lipid nanoparticles comprising the compound of structural formula (I) and a therapeutic agent and/or a prophylactic agent, and delivering the composition to a subject.

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HUANG CAIGU (CN)
HUAGN TIEQIANG (CN)

PCT/CN2022/084244

February 16, 2023

March 31, 2022

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GUAGNGZHOU ANOVENT PHARMACEUTICAL CO LTD (CN)

C07C229/16; A61K9/127; A61K9/51; A61K47/14; C07C219/06; C12N15/11

WO2021050864A1	2021-03-18
WO2020061367A1	2020-03-26
WO2018144775A1	2018-08-09

CN110520409A	2019-11-29
CN108368028A	2018-08-03

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