Cationic Lipid Structure for Nucleic Acid Delivery Efficiency

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Solution Overview

Problem

Current cationic lipids do not efficiently introduce nucleic acids into various cells, tissues, or organs, limiting the effectiveness and safety of nucleic acid drugs.

Innovation Solution

A compound represented by the formula, specifically 3-((4-(Dimethylamino)butanoyl)oxy)-2,2-bis(((9Z)-tetradec-9-enoyloxy)methyl)propyl (9Z)-tetradec-9-enoate or its salts, is used to form lipid particles that facilitate the introduction of nucleic acids into cells, tissues, or organs with high efficiency.

Engineering Contradictions & Design Principles

VSEngineering Contradiction Analysis

1Reliability

If conventional cationic lipids are used to form complexes with nucleic acids, then the nucleic acids can be introduced into cells, but the introduction efficiency is insufficient and cannot satisfy the requirements for high efficiency and safety across various cell types

Engineering Contradiction:
Improvenucleic acid introduction efficiencyVSAvoidapplicability to various cell types
Core Design Contradiction:
ReliabilityVSAdaptability or versatility

Solution Approach 1:

The patent modifies the chemical structure of cationic lipids by changing parameters such as the hydrocarbon chain length (C16-C24), the presence of double bonds (0-3 double bonds), and the substituent groups (R1-R6, Y1-Y3) to create a series of lipid variants with optimized properties for different cell types and nucleic acid delivery requirements

Inventive Principle:
Principle #35Parameter changes

Solution Approach 2:

The patent creates composite lipid-nucleic acid complexes where the cationic lipid structure is specifically designed to interact with nucleic acids through electrostatic interactions, forming stable complexes that can be efficiently taken up by various cell types while maintaining the biological activity of the nucleic acid

Inventive Principle:
Principle #40Composite materials

Applied Scientific Principles

This section explains which scientific principles are used to turn an abstract innovation direction into a practical engineering solution.

Function Achieved in This Case

The compound enables superior efficiency in introducing nucleic acids into various types of cells, tissues, or organs, enhancing the manifestation of drug action and safety of nucleic acid drugs.

Implementation Method 1

a nucleic acid and a lipid are mixed to form a complex

Methodology Applied
Scientific EffectElectrostatic interaction: Electrostatics

Data Source

PatentUS11993570B2Cationic lipids
Publication Date: 2024.05.28 TAKEDA PHARMA CO LTD
  • US11993570B2 patent drawing
  • US11993570B2 patent drawing
  • US11993570B2 patent drawing

AI summary

The present invention provides a technique that enables introduction of active ingredients, in particular, nucleic acids, into cells with superior efficiency; and cationic lipids, etc., for use in the technique. The compound or a salt thereof according to the present invention is a compound represented by formula (I) or a salt thereof. In formula (I), n represents an integer of 2 to 5, R represents a linear C1-5 alkyl group, a linear C7-11 alkenyl group, or a linear C11 alkadienyl group, and wavy lines each independently represent a cis-bond or a trans-bond.