Templated Assembly Reactants for Targeted Pathogenic Cell Elimination
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Solution Overview
Problem
Current bio-therapeutic interventions face challenges in effectively targeting and eliminating pathogenic cells without causing toxicity to normal cells, due to limited availability of target antigens and inefficiencies in down-modulating pathogenic genes or eliminating pathogenic cells.
Innovation Solution
The development of targeted templated assembly reactants that include a nucleic acid recognition moiety, a selectively-reactive moiety, and an effector partial moiety, which bind specifically to target nucleic acid sequences in pathogenic cells to produce an active effector structure that induces desired biological activities such as cell death or gene expression modulation.
Engineering Contradictions & Design Principles
Engineering Contradiction Analysis
1Reliability
If existing nucleic acid-targeted therapies (such as siRNA) are used to down-modulate expression of pathogenic genes, then gene expression can be reduced, but the therapies cannot deliver potent cytotoxic or cytostatic interventions and are not efficient at eliminating pathogenic cells
Solution Approach 1:
The therapeutic agent is divided into separate modular components: a nucleic acid recognition moiety that binds to pathogenic cell-specific sequences, a selectively-reactive moiety that remains dormant during delivery, and an effector partial moiety that is activated only after cellular uptake and templated assembly, enabling potent cytotoxic activity while maintaining delivery safety
Solution Approach 2:
The nucleic acid recognition moiety is designed to bind to pathogenic cell-specific nucleic acid sequences during delivery, pre-positioning the selectively-reactive moiety for subsequent activation. This preliminary binding ensures that the effector is only activated in the correct cellular context, improving both reliability and efficiency of pathogenic cell elimination
2Reliability
If targeted therapeutics are developed to deliver potent interventions to pathogenic cells, then cell elimination can be achieved, but it is extremely difficult to discover protein markers for many pathogenic cell types
Solution Approach 1:
The patent replaces protein marker-based targeting (which requires complex discovery and validation) with nucleic acid sequence-based targeting. Nucleic acid sequences can be discovered through straightforward genomic and transcriptomic analysis, significantly easing the marker discovery process while maintaining high targeting accuracy through sequence-specific binding
Solution Approach 2:
The nucleic acid recognition moiety can be designed to target any pathogenic cell type by simply changing the bound nucleic acid sequence, making the platform universally applicable across different diseases and cell types without requiring development of new targeting mechanisms for each application
3Reliability
If potent bio-therapeutic interventions are delivered to pathogenic cells, then pathogenic cells can be eliminated, but toxicity to adjacent normal cells or overall patient health occurs
Solution Approach 1:
The effector is designed to be locally activated only within pathogenic cells that contain the specific target nucleic acid sequence. The templated assembly mechanism ensures that the selectively-reactive moiety is only activated in the presence of the correct nucleic acid template, creating local quality control that prevents systemic toxicity to normal cells
Solution Approach 2:
The nucleic acid recognition moiety acts as an intermediary that specifically binds to pathogenic cell sequences, serving as a selective gateway that directs the effector exclusively to pathogenic cells. This intermediary mechanism ensures high-fidelity targeting and prevents off-target effects on normal cells
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
This approach allows for precise targeting of pathogenic cells, reducing off-target toxicity and enhancing pathogen-specific reactivity, enabling effective elimination of pathogenic cells while minimizing harm to normal cells.
Implementation Method 1
at least one nucleic acid recognition moiety that binds a target nucleic acid sequence
Data Source
AI summary
The present disclosure is directed methods and products for synthesizing and using targeted templated assembly reactants comprising at least one nucleic acid recognition moiety, at least one selectively-reactive moiety, and at least one effector partial moiety. The nucleic acid recognition moiety can bind a target nucleic acid sequence within a sample. The nucleic acid recognition moiety also can bind the selectively-reactive moiety. Additionally, the effector partial moiety can bind the selectively-reactive moiety to produce an active effector structure. Also disclosed are methods of delivering the targeted templated assembly reactants and active effector structures formed from the targeted templated assembly reactants.