PolyC:Poly(G/I) dsRNA TLR3 Agonist Stability

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Solution Overview

Problem

Current TLR3 agonists, such as poly(I:C), have undefined chemical structures, poor homogeneity, and toxicity issues due to rapid degradation in body fluids, limiting their effectiveness in activating immune cells and inducing apoptosis in cancer cells.

Innovation Solution

Development of double-stranded RNAs (dsRNAs) with a defined chemical composition and molecular weight, comprising one strand of poly(C) and a complementary strand with guanosine (G) and inosine (I) residues, optimized for solubility and potency in triggering innate immunity through TLR3 and RIG-I pathways.

Engineering Contradictions & Design Principles

VSEngineering Contradiction Analysis

1Reliability

If poly(I:C) is used as a TLR3 agonist, then immune cells are activated, but the chemical structure is undefined and homogeneity is poor

Engineering Contradiction:
Improveimmune cell activationVSAvoidchemical structure definition
Core Design Contradiction:
ReliabilityVSManufacturing precision

Solution Approach 1:

The patent changes the chemical parameters of the dsRNA by substituting inosine residues for specific positions in the poly(I:C) sequence. This modification creates a defined chemical structure with specific homogeneity while maintaining the TLR3 agonist activity. The inosine substitutions at defined positions provide structural precision without compromising immune activation capability.

Inventive Principle:
Principle #35Parameter changes

2Reliability

If poly(I:C) is used to activate immune cells, then immune response is induced, but toxicity occurs due to rapid degradation in body fluids

Engineering Contradiction:
Improveimmune response inductionVSAvoidtoxicity from degradation
Core Design Contradiction:
ReliabilityVSObject-affected harmful factors

Solution Approach 1:

The patent applies local quality modification by introducing inosine residues at specific positions within the dsRNA sequence rather than uniformly throughout. This localized modification at defined positions enhances stability and reduces degradation-related toxicity while preserving the essential immune activation function at other regions of the molecule.

Inventive Principle:
Principle #3Local quality

3Reliability

If poly(I:C) is used for cancer treatment, then apoptosis is induced in tumor cells, but the structure is heterogeneous

Engineering Contradiction:
Improveapoptosis inductionVSAvoidstructural homogeneity
Core Design Contradiction:
ReliabilityVSStability of the object's composition

Solution Approach 1:

The patent modifies the compositional parameters of the dsRNA by incorporating inosine at specific positions defined in the sequence. This creates a homogeneous composition with precise chemical definition while maintaining the apoptotic activity in tumor cells. The defined inosine substitutions ensure consistent structural composition across all molecules.

Inventive Principle:
Principle #35Parameter changes

4Reliability

If poly(I:C) is used as a TLR3 agonist, then immune activation occurs, but solubility is limited requiring heating and slow cooling

Engineering Contradiction:
ImproveTLR3 agonist activityVSAvoidsolubility and reconstitution
Core Design Contradiction:
ReliabilityVSEase of manufacture

Solution Approach 1:

The patent changes the physicochemical parameters of the dsRNA by introducing inosine residues at specific positions. This modification improves solubility characteristics, allowing the molecule to dissolve without requiring heating and slow cooling procedures. The defined inosine substitutions alter the molecular properties to enhance ease of manufacture and reconstitution.

Inventive Principle:
Principle #35Parameter changes

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 dsRNAs demonstrate enhanced potency in activating immune cells and inducing apoptosis in cancer cells, with improved stability and solubility compared to prior art, achieving stronger immune responses and specific pro-apoptotic activity on tumor cells.

Implementation Method 1

double-stranded ribonucleic acids (dsRNAs) as defined in claim 1 for triggering innate immunity, in particular through toll-like receptor 3 (TLR3) and, optionally, RIG-I or RIG-I-like receptors (RLRs)

Methodology Applied
Scientific EffectToll-like receptor 3 (TLR3) recognition:

Implementation Method 2

double-stranded ribonucleic acids (dsRNAs) as defined in claim 1 for triggering innate immunity, in particular through toll-like receptor 3 (TLR3) and, optionally, RIG-I or RIG-I-like receptors (RLRs)

Methodology Applied
Scientific EffectRIG-I receptor recognition:

Data Source

PatentEP3083962B1Double-stranded polyc:poly(g/i) RNA for immunostimulation and cancer treatment
Publication Date: 2021.08.18 RIBOXX
  • EP3083962B1 patent drawingFigure 1
  • EP3083962B1 patent drawingFigure 2A~2B
  • EP3083962B1 patent drawingFigure 3

AI summary

The present invention relates to polyC:poly(G/l) dsRNAs for triggering innate immunity, in particular through toll-like receptor 3 (TLR-3) and, optionally, RIG-I or RIG-I— like receptors (RLRs), as well as compositions and medicaments containing such dsRNAs, methods for their production and their use in medicine, especially immunostimulation and prevention and/or therapy of infections and tumor diseases.