Lipid Particle Composition for High Nucleic Acid Encapsulation
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Solution Overview
Problem
Existing nucleic acid delivery methods, such as viral vectors, face limitations in gene size transfer and immunogenicity concerns, while lipid particles offer a solution but require compounds that enhance nucleic acid encapsulation and delivery efficiency.
Innovation Solution
Development of compounds represented by Formula (1) or their salts, which are incorporated into lipid particles, achieving high nucleic acid encapsulation rates and efficient delivery.
Engineering Contradictions & Design Principles
Engineering Contradiction Analysis
1Productivity
If viral vectors are used for nucleic acid delivery, then gene transfer efficiency is high, but gene size transfer is limited and immunogenicity concerns arise
Solution Approach 1:
The patent introduces lipid particles as an intermediary carrier system that can accommodate nucleic acids of various sizes without the immunogenicity issues of viral vectors. The lipid particles serve as a non-viral mediator that delivers genetic material while avoiding the harmful effects associated with viral-based systems.
Solution Approach 2:
The patent modifies the chemical structure of lipid particles by incorporating specific compounds (Formula 1) with particular molecular weights and structural features. These parameter changes in the lipid particle composition enable enhanced encapsulation efficiency and delivery capability while maintaining versatility for different gene sizes.
2Adaptability or versatility
If lipid particles are used for nucleic acid delivery, then gene size limitation is overcome, but nucleic acid encapsulation rate and delivery efficiency need improvement
Solution Approach 1:
The patent optimizes the chemical parameters of the lipid particle composition by incorporating compounds with specific molecular weights (500-5000 Da) and structural features (Formula 1). These parameter changes enhance the encapsulation rate and delivery efficiency while maintaining the ability to accommodate various gene sizes.
Solution Approach 2:
The patent creates a composite lipid particle system combining multiple lipid components with specific compounds (Formula 1). This composite approach leverages the synergistic effects of different materials to achieve high encapsulation rates and efficient delivery while maintaining versatility for different nucleic acid sizes.
3Ease of operation
If existing lipid particle compounds are used, then nucleic acid delivery is achieved, but encapsulation rate and delivery efficiency are insufficient
Solution Approach 1:
The patent systematically optimizes the chemical parameters of the lipid particle formulation by testing and selecting compounds with specific molecular weights and structural features (Formula 1). These parameter optimizations result in significant improvements in both encapsulation rate and delivery efficiency while maintaining ease of operation.
Data Source
AI summary
An object of present invention is to provide a compound or a salt thereof constituting lipid particles that can achieve a high nucleic acid encapsulation rate and excellent delivery of nucleic acids, and to provide lipid particles and pharmaceutical composition using the compound or a salt thereof, which can achieve a high nucleic acid encapsulation rate and excellent delivery of nucleic acids. According to an aspect of the present invention, a compound represented by Formula (1) or a salt thereof is provided. In the formula, each symbol has a meaning defined in the present specification.