Novel cationic lipid compounds

The present invention relates to lipid compounds of the structure of formula (I), which can be used alone or in combination with other lipid components such as neutral lipids, spot lipids, steroids and/or analogs thereof, and/or polymer-conjugated lipids, to form lipid nanoparticles for the delivery...

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Hauptverfasser: WANG SHUAI, HUANG CAIGU
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HUANG CAIGU
description The present invention relates to lipid compounds of the structure of formula (I), which can be used alone or in combination with other lipid components such as neutral lipids, spot lipids, steroids and/or analogs thereof, and/or polymer-conjugated lipids, to form lipid nanoparticles for the delivery of therapeutic and/or prophylactic agents. In some examples, lipid nanoparticles are used to deliver nucleic acids, such as messenger RNA and/or antisense RNA. Methods of using such lipid nanoparticles to treat and/or prevent various diseases are also provided. Wherein the structure of the formula (I) is as shown in the specification, or salt or isomer or N-oxide thereof, and R1 is as defined in the specification. 本发明涉及式(I)结构的脂质化合物,其可以单独使用或者与其他脂质组分例如中性脂质、带点脂质、类固醇和/或它们的类似物、和/或聚合物缀合的脂质联合使用,以形成用于递送治疗剂和/或预防剂的脂质纳米颗粒。在一些实例中,将脂质纳米颗粒用于递送核酸,例如信使RNA和/或反义RNA。还提供了使用这类脂质纳米颗粒来治疗和/或预防各种疾病的方法。其中式(I)结构如下:或其盐或异构体或其N-氧化物,其中R1如文中所定义。
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In some examples, lipid nanoparticles are used to deliver nucleic acids, such as messenger RNA and/or antisense RNA. Methods of using such lipid nanoparticles to treat and/or prevent various diseases are also provided. 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subjects ACYCLIC OR CARBOCYCLIC COMPOUNDS
CHEMISTRY
HUMAN NECESSITIES
HYGIENE
MEDICAL OR VETERINARY SCIENCE
METALLURGY
ORGANIC CHEMISTRY
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
title Novel cationic lipid compounds
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