RNA-Encoded Docking Compounds for Targeted Cell Delivery
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Solution Overview
Problem
Current methods for targeted delivery of therapeutic or diagnostic agents lack specificity and efficiency in reaching target cells, such as cancer cells, due to non-selective binding and distribution within the body.
Innovation Solution
The use of RNA-encoded docking compounds that express peptide or polypeptide binding moieties, allowing for targeted binding to specific cell surface antigens, followed by the delivery of a payload linked to a secondary binding moiety that binds to the docking compound, enabling precise delivery to target cells.
Engineering Contradictions & Design Principles
Engineering Contradiction Analysis
1Measurement precision
If conventional targeted delivery methods are used, then delivery to target cells is attempted, but specificity and efficiency are insufficient due to non-selective binding and distribution
Solution Approach 1:
The delivery system is divided into two separate components: a targeting moiety that binds specifically to target cells and a payload that delivers the therapeutic or diagnostic agent. This segmentation allows each component to be optimized independently for its specific function, improving both specificity of targeting and efficiency of payload delivery without compromising the other.
Solution Approach 2:
The patent introduces a docking compound as an intermediary component that serves as a bridge between the targeting moiety and the payload. The docking compound contains a binding moiety that specifically recognizes and binds to target cells, and a secondary binding moiety that interacts with the payload. This intermediary structure enables precise control over the delivery process, ensuring that payloads are delivered only to cells that express the specific target antigen, thereby resolving the contradiction between specificity and delivery efficiency.
2Productivity
If non-specific binding is used for delivery, then distribution throughout the body occurs, but precision in reaching target cells is reduced
Solution Approach 1:
The targeting moiety is designed with specific local properties that enable it to recognize and bind to particular antigens expressed on target cells. By conferring this specific binding capability to the delivery system, the patent ensures that payloads are concentrated at the target site rather than being distributed non-specifically throughout the body, thus achieving both high delivery efficiency and high targeting precision.
Solution Approach 2:
The docking compound acts as a mediator that translates the specific recognition of target cells into efficient payload delivery. The binding moiety of the docking compound provides the specificity for target cell recognition, while the secondary binding moiety ensures efficient coupling to and delivery of the payload. This mediating structure resolves the contradiction by ensuring that high delivery efficiency is achieved only at sites of specific target recognition.
Data Source
AI summary
The invention relates to agents and methods for targeted delivery of payloads to cells. The agents and methods are useful for delivering therapeutic or diagnostic agents to target cells. In one embodiment, the invention involves administering RNA encoding a peptide or polypeptide (docketing compound) comprising a binding moiety (primary targeting moiety) binding to target cells and a further binding moiety (secondary target) binding to an agent that comprises a payload (effector probe). Following expression of the RNA, the primary targeting moiety may bind to a target antigen such as a cancer antigen on cancer cells and then a secondary targeting moiety comprised in the effector probe may target the secondary target to thereby precisely deliver a “payload” to the target cells such as cancer cells.