Modular Nucleic Acid Nanoparticles for Stable Cargo Delivery
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Solution Overview
Problem
Current nucleic acid nanoparticle therapies face challenges in delivering therapeutic agents to specific cells intact, internalizing them, and maintaining activity at target sites while avoiding off-target effects, requiring extensive redesign for each application.
Innovation Solution
Nanoparticles with modular design using bioorthogonal click chemistry for stable scaffolds and functional regulation, allowing interchangeable attachment of cargo molecules via various linkages, enhancing stability, reproducibility, and therapeutic payload.
Engineering Contradictions & Design Principles
Engineering Contradiction Analysis
1Reliability
If nucleic acid nanoparticles are designed specifically for each indication, then therapeutic efficacy is improved, but device complexity and development time increase
Solution Approach 1:
The nanoparticle system is divided into modular components: a stable nucleic acid core scaffold and interchangeable cargo molecules. This segmentation allows the core structure to remain constant while only the cargo portion needs to be changed for different therapeutic indications, reducing overall design complexity while maintaining efficacy.
Solution Approach 2:
The nucleic acid nanoparticle core is designed as a universal platform that can deliver multiple types of cargo molecules including siRNA, mRNA, and small molecule drugs. This multi-functionality allows a single core design to serve multiple therapeutic indications, eliminating the need to redesign the entire nanoparticle for each application.
2Adaptability or versatility
If nucleic acid nanoparticles are redesigned for each application, then adaptability to different indications is improved, but manufacturing precision and reproducibility decrease
Solution Approach 1:
The stable nucleic acid core scaffold is pre-designed and optimized for stability and delivery functionality before cargo attachment. This preliminary action establishes a consistent foundation that ensures reproducible manufacturing across different therapeutic applications, as only the cargo portion requires modification rather than the entire structure.
3Reliability
If cargo molecules are attached via traditional methods, then delivery function is achieved, but stability and activity preservation are insufficient
Solution Approach 1:
Functional linkers serve as intermediaries between the nucleic acid core scaffold and cargo molecules. These linkers enable stable attachment while preserving cargo activity, and they facilitate simplified attachment processes through standardized interaction interfaces, resolving the contradiction between stability and ease of manufacture.
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 modular design increases cargo capacity, reduces development time, and enhances therapeutic efficacy by enabling multiple functionalities and synergistic effects, while minimizing off-target effects.
Implementation Method 1
therapies based on nucleic acid nanoparticles are plagued by a variety of problems that have yet to be overcome. Many promising therapeutic agents are biological macromolecules that need to be delivered intact to the right cells in the body, internalized by those cells
Implementation Method 2
The nanoparticle compositions have a modular design, which is achieved via the use of bioorthogonal click chemistry (FIG. 1). These reactions are used for both ligation and stabilisation.
Data Source
AI summary
The invention provides compositions containing cargo molecules attached to elements that improve the function of the cargo molecules in the body of a subject. The compositions are useful for therapeutic and diagnostic purposes. Furthermore, the invention outlines ways in which these compositions can be produced; the core molecule can be functionalized, via bioorthogonal click chemistry, in such a way as to impart modular characteristics. This functionalization simultaneously allows for loading of biologically relevant cargo and provides stabilization to the overall structure of the molecule.