Novel Lipid Compound for High-Efficiency Nucleic Acid Encapsulation
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Solution Overview
Problem
Existing lipid compositions for delivering nucleic acids into cells face limitations in encapsulation efficiency and delivery efficacy, particularly when using non-viral vectors, and there is a need for compounds that can enhance these processes.
Innovation Solution
A compound represented by Formula (1) or its salt, which includes specific hydrocarbon groups and linking moieties, is used to formulate a lipid composition that achieves high nucleic acid encapsulation and efficient delivery.
Engineering Contradictions & Design Principles
Engineering Contradiction Analysis
1Productivity
If a viral vector is used for gene introduction, then high gene introduction efficiency is achieved, but the size of genes to be introduced is limited and immunogenicity and safety concerns arise
Solution Approach 1:
The patent uses a lipid composition as a disposable, non-viral delivery system that can be synthesized chemically and used for gene introduction without the need for viral vectors. This approach allows for flexible gene size accommodation and avoids immunogenicity issues associated with viral vectors, while maintaining effective delivery capability through the lipid-nucleic acid complex formation
2Adaptability or versatility
If conventional lipid compositions are used for nucleic acid delivery, then non-viral delivery is achieved, but encapsulation efficiency and delivery efficacy are limited
Solution Approach 1:
The patent modifies the lipid composition by incorporating specific compounds with defined chemical structures (Formula 1 and Formula 2) that have optimized hydrophobic and hydrophilic regions. This parameter change in molecular structure enhances the lipid's ability to encapsulate nucleic acids efficiently while maintaining delivery efficacy, resolving the limitation of conventional lipid compositions
Data Source
AI summary
An object of the present invention is to provide a compound or a salt thereof constituting lipid composition that can achieve a high nucleic acid encapsulation rate and excellent delivery of nucleic acids, and to provide a lipid composition, a pharmaceutical composition, and a delivery carrier, using the compound or a salt thereof. According to the present invention, a compound represented by Formula (1) or a salt thereof is provided. In the formula, R1, R2, R3, and R4 each independently represent a hydrogen atom or a hydrocarbon group having 1 to 24 carbon atoms, which may be substituted, R5 and R6 each independently represent a hydrocarbon group having 1 to 18 carbon atoms, which may be substituted, R7, R8, and R9 each independently represent a hydrocarbon group having 2 to 8 carbon atoms, and R5 and R6, or R5 and R7, may be combined to form a 4- to 7-membered ring.