Biodegradable Dendrimer Carriers for Stable Nucleic Acid Release
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Solution Overview
Problem
Existing nucleic acid delivery systems, such as polyester dendrimers and disulfide compounds, face challenges in efficiently delivering nucleic acids due to poor stability and potential in vivo side reactions, necessitating the development of compounds that can efficiently deliver nucleic acids and degrade to low molecular weight compounds at the desired site of action.
Innovation Solution
The use of certain dendrimers and dendrons with specific structural features, including disulfide, heteroaryl, or C1-C6 alkylene cores, and various linkages, which are highly biodegradable and efficient in delivering nucleic acids, forming nanoparticle compositions that can encapsulate therapeutic or immunogenic nucleic acids.
Engineering Contradictions & Design Principles
Engineering Contradiction Analysis
1Duration of action of moving object
If disulfide compounds are used in delivery systems, then controlled release of therapeutic cargos is enabled, but stability is poor in the presence of reducing agents and potential in vivo side reactions occur
Solution Approach 1:
The patent modifies the disulfide linkage structure by introducing specific substituents and varying the molecular architecture of the dendrimer/dendron carriers. This changes the chemical environment and steric protection around the disulfide bond, making it more resistant to premature reduction while maintaining controlled release capability at the target site.
Solution Approach 2:
The invention combines disulfide linkages with polyester dendrimer or dendron structures to create composite delivery systems. The polyester framework provides structural stability and biodegradability, while the disulfide linkage enables controlled release, creating a synergistic material that balances both stability and controlled release functionality.
2Productivity
If biodegradable linkages are positioned to enhance payload release, then degradation efficiency improves, but overall stability and ability to produce surviving nanoparticles deteriorates
Solution Approach 1:
The patent introduces biodegradable linkages at specific local positions within the dendrimer/dendron structure rather than uniformly throughout. By strategically placing cleavable bonds at terminal or specific internal positions, the design enables efficient payload release at the target site while maintaining the structural integrity and stability of the core nanoparticle framework during circulation.
Solution Approach 2:
The invention divides the delivery system into distinct functional segments: a stable core structure for circulation and targeting, and biodegradable peripheral linkages for controlled release. This segmentation allows different parts of the molecule to have different stability characteristics, with the core providing overall stability and the peripheral linkages providing controlled degradation for payload release.
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
These compounds effectively deliver nucleic acids to targeted cells while degrading to low molecular weight compounds, enhancing payload release and stability in vivo, thereby improving therapeutic efficacy.
Implementation Method 1
certain dendrimers and dendrons are highly biodegradable and also very efficient in delivering nucleic acids
Data Source
AI summary
Disclosed are compounds useful for delivering therapeutic or immunogenic nucleic acid agents. Further disclosed are nanoparticle compositions having such a compound and methods for treating or preventing a disease or condition.