Ionizable Lipid Nanoparticles for High-Efficiency RNA Encapsulation

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Solution Overview

Problem

Existing lipid-based nanoparticles face challenges in effectively loading and delivering therapeutic and diagnostic compounds to specific locations in the body, particularly for hydrophilic agents like DNA and RNA, necessitating the development of superior ionizable lipids for enhanced drug delivery.

Innovation Solution

Development of novel ionizable lipids represented by specific chemical structures that spontaneously form nanoparticles, capable of encapsulating active agents and delivering them to target sites with high encapsulation efficiency and cellular internalization.

Engineering Contradictions & Design Principles

VSEngineering Contradiction Analysis

1Reliability

If conventional lipid-based nanoparticles are used for delivering hydrophilic agents like DNA and RNA, then the delivery system can be established, but the encapsulation efficiency is insufficient and dosing is not optimized

Engineering Contradiction:
Improveencapsulation efficiencyVSAvoiddelivery effectiveness
Core Design Contradiction:
ReliabilityVSProductivity

Solution Approach 1:

The patent modifies the chemical structure of ionizable lipids by changing parameters such as introducing specific functional groups (carboxylic acid, hydroxyl, amino, or carbonyl groups) at defined positions in the lipid molecule. These structural parameter changes enable the lipids to achieve optimal encapsulation efficiency (at least 30%) and intracellular expression (10 times greater than existing methods) for hydrophilic agents like DNA and RNA, thereby resolving the contradiction between reliability and productivity.

Inventive Principle:
Principle #35Parameter changes

2Reliability

If existing ionizable lipids are used, then some encapsulation is achieved, but the ability to deliver active agents to specific locations in the body is limited

Engineering Contradiction:
Improvetargeted delivery capabilityVSAvoidspecific location delivery
Core Design Contradiction:
ReliabilityVSAdaptability or versatility

Solution Approach 1:

The patent introduces ionizable lipid molecules with specific local chemical properties (ionizable functional groups with controlled pKa values between 5-9) that enable selective interaction with target cells at specific locations in the body. The lipid structure includes localized functional regions that facilitate endosomal escape and intracellular delivery, providing targeted delivery capability while maintaining adaptability for different therapeutic agents.

Inventive Principle:
Principle #3Local quality

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 ionizable lipids achieve at least 30% encapsulation efficiency and 10 times greater intracellular expression of RNA compared to existing methods, facilitating targeted delivery of therapeutic and diagnostic agents.

Implementation Method 1

ionizable lipids and lipid nanoparticles comprising same

Methodology Applied
Scientific EffectIonization: Ionisation

Implementation Method 2

nanoparticles comprising same... capable of encapsulating active agents

Methodology Applied
Scientific EffectSelf-assembly: Self-Assembly

Data Source

PatentUS20250352488A1Ionizable lipids and nanoparticles comprising same
Publication Date: 2025.11.20 MANA BIO LTD
  • US20250352488A1 patent drawing
  • US20250352488A1 patent drawing
  • US20250352488A1 patent drawing

AI summary

One or more ionizable lipid(s) and lipid nanoparticles comprising same are provided. Pharmaceutical compositions comprising the lipid nanoparticles encapsulating an active agent are also provided.