Cationic Lipid Phase Transition for Nucleic Acid Delivery

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Solution Overview

Problem

Conventional cationic lipids used in lipid membrane structures for nucleic acid delivery face challenges in achieving high intracellular delivery efficiency due to poor membrane fusion ability and pH responsiveness, leading to low release of functional nucleic acids into the cytoplasm.

Innovation Solution

A cationic lipid with a hydrophobic moiety containing 1 to 4 hydrophobic groups and a hydrophilic moiety with one or two cationic groups is developed, allowing for phase transition from a lamellar to an inverted hexagonal phase under acidic conditions, enhancing membrane fusion ability and nucleic acid delivery.

Engineering Contradictions & Design Principles

VSEngineering Contradiction Analysis

1Reliability

If conventional two-chain type cationic lipids are used in lipid membrane structures, then the carrier can be stably present in physiological environment, but the membrane fusion ability is poor and intracellular delivery efficiency is low

Engineering Contradiction:
Improvestability in physiological environmentVSAvoidintracellular delivery efficiency
Core Design Contradiction:
ReliabilityVSProductivity

Solution Approach 1:

The patent modifies the molecular structure of cationic lipids by changing the hydrophobic moiety configuration - using one-chain type with specific chain lengths and compositions instead of conventional two-chain types. This structural parameter change enables the lipid to undergo phase transition from lamellar to inverted hexagonal phase under acidic conditions, dramatically improving membrane fusion ability while maintaining stability in physiological conditions through careful selection of hydrophobic chain characteristics.

Inventive Principle:
Principle #35Parameter changes

Solution Approach 2:

The invention exploits the phase transition property of the modified cationic lipid, which transitions from a lamellar phase at physiological pH to an inverted hexagonal phase under acidic conditions in endosomes. This phase transition mechanism enables the lipid membrane structure to fuse with endosomal membranes and release functional nucleic acids into the cytoplasm, resolving the contradiction between stability and delivery efficiency.

Inventive Principle:
Principle #36Phase transitions

2Stability of the object's composition

If conventional cationic lipids are used, then the lipid membrane structure can be formed, but the release of functional nucleic acids into cytoplasm is insufficient

Engineering Contradiction:
Improvelipid membrane structure formationVSAvoidamount of nucleic acid released into cytoplasm
Core Design Contradiction:
Stability of the object's compositionVSQuantity of substance

Solution Approach 1:

The patent changes the chemical structure parameters of the cationic lipid, specifically using one-chain type cationic lipids with defined hydrophobic chain lengths and compositions. This structural modification enables the lipid to form stable membrane structures that can undergo acid-induced phase transition, thereby releasing significantly higher amounts of functional nucleic acids into the cytoplasm compared to conventional lipids.

Inventive Principle:
Principle #35Parameter changes

Solution Approach 2:

The invention utilizes the acid-induced phase transition from lamellar to inverted hexagonal phase as a trigger mechanism for nucleic acid release. The modified cationic lipid maintains structural integrity at physiological pH but transitions to a non-lamellar phase under acidic endosomal conditions, enabling efficient release of encapsulated functional nucleic acids into the cytoplasm.

Inventive Principle:
Principle #36Phase transitions

3Adaptability or versatility

If amino groups in cationic lipid are protonated to impart pH responsiveness, then the lipid membrane structure shows pH responsiveness, but the hydrophilic moiety expands and repels itself, preventing phase transition

Engineering Contradiction:
ImprovepH responsivenessVSAvoidmembrane phase structure
Core Design Contradiction:
Adaptability or versatilityVSShape

Solution Approach 1:

The patent modifies the molecular architecture of the cationic lipid by using a one-chain type structure with specific hydrophobic chain characteristics. This structural parameter change reduces the expansion of the hydrophilic moiety upon protonation, allowing the lipid to undergo phase transition from lamellar to inverted hexagonal phase under acidic conditions while maintaining pH responsiveness for targeted release.

Inventive Principle:
Principle #35Parameter changes

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 new cationic lipid formulation achieves high membrane fusion ability under acidic conditions, resulting in efficient release of functional nucleic acids into the cytoplasm, significantly improving gene knockdown efficacy compared to conventional two-chain type cationic lipids.

Implementation Method 1

allowing for phase transition from a lamellar to an inverted hexagonal phase under acidic conditions

Methodology Applied
Scientific EffectPhase transition: Phase Change

Implementation Method 2

The amino group contained in the cationic lipid is protonated and changes to cationic as the surrounding pH decreases, thereby imparting pH responsiveness to the lipid membrane structure

Methodology Applied
Scientific EffectProtonation:

Data Source

PatentEP3184506B1Cationic lipid for nucleic acid delivery
Publication Date: 2019.07.24 NOF CORP
  • EP3184506B1 patent drawingFigure 1~2
  • EP3184506B1 patent drawingFigure 3~4
  • EP3184506B1 patent drawingFigure 5~6

AI summary

An object of the present invention is to provide a cationic lipid capable of achieving higher intracellular delivery efficiency than conventional cationic lipids, when used as a lipid membrane structure which is a carrier for delivering functional nucleic acid. A cationic lipid represented by the formula (1): wherein each symbol is as defined in the present DESCRIPTION.