Multigene Editing Vector for Cancer Therapy

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Solution Overview

Problem

Current cancer therapies are limited in effectively addressing multigenic diseases like cancer due to the complexity of multiple genetic abnormalities, undruggable targets, and resistance mechanisms, with existing gene editing technologies exhibiting low efficiency and limited to single gene locus modifications.

Innovation Solution

The development of a vector comprising a donor therapeutic mRNA, a genome editing endonuclease, and guide RNA sequences that simultaneously disrupt pathogenic genes and express therapeutic moieties, allowing for concurrent inhibition of multiple targets in a single treatment, including undruggable targets, and reversing treatment resistance.

Engineering Contradictions & Design Principles

VSEngineering Contradiction Analysis

1Reliability

If multiple therapies are used to address multiple genetic abnormalities in cancer, then treatment efficacy is improved, but cumulative toxicities in normal tissues increase

Engineering Contradiction:

Improvetreatment efficacyVSAvoidcumulative toxicities

Core Design Contradiction:

ReliabilityVSObject-affected harmful factors

Solution Approach 1:

The patent combines multiple therapeutic functions into a single vector system that can simultaneously deliver genome editing endonucleases and therapeutic genes to multiple target loci, achieving the benefits of combination therapy while reducing cumulative toxicities by consolidating treatment delivery

Inventive Principle:

Principle #5Merging (Combining)

Solution Approach 2:

The vector system is designed with multi-functionality to perform both genome editing and therapeutic gene delivery simultaneously, allowing a single treatment approach to address multiple genetic abnormalities without requiring separate therapeutic agents

Inventive Principle:

Principle #6Universality (Multi-functionality)

2Reliability

If current gene editing technologies are used to modify single gene loci, then genetic corrections can be achieved, but treatment efficiency remains low (typically 5% or less)

Engineering Contradiction:

Improvegenetic correction capabilityVSAvoidgene correction efficiency

Core Design Contradiction:

ReliabilityVSProductivity

Solution Approach 1:

The patent segments the genome editing function into modular components (endonuclease, guide RNA, donor template) that can be independently optimized and combined, enabling systematic improvement of editing efficiency across multiple loci simultaneously

Inventive Principle:

Principle #1Segmentation

Solution Approach 2:

The patent introduces donor therapeutic mRNA and DNA templates as intermediary elements that facilitate more efficient gene correction by providing pre-prepared repair templates that guide the editing process, thereby increasing correction efficiency beyond current standards

Inventive Principle:

Principle #24Intermediary (Mediator)

3Adaptability or versatility

If conventional methods are used to target undruggable molecular targets, then comprehensive cancer pathway coverage can be achieved, but resistance mechanisms are triggered

Engineering Contradiction:

Improvemolecular target coverageVSAvoidtreatment resistance

Core Design Contradiction:

Adaptability or versatilityVSReliability

Solution Approach 1:

The patent replaces conventional small molecule drug mechanisms with genome editing and gene therapy mechanisms, enabling direct modification of genetic pathways rather than relying on drug-receptor interactions that are susceptible to resistance development

Inventive Principle:

Principle #28Mechanics substitution (Replace mechanical system)

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 enables an unprecedented ability to address multiple molecular abnormalities in multigenic diseases, achieving synergistic effects and overcoming the limitations of current therapies by providing an efficacious and well-tolerated in vivo gene editing therapy.

Implementation Method 1

Genome editing approaches involve the use of meganucleases, zinc finger nucleases (ZFNs), transcription activator-like effector-based nucleases (TALEN), and the clustered regularly interspaced short palindromic repeats (CRISPR/Cas) systems

Methodology Applied

Scientific EffectCRISPR/Cas genome editing: