Oligonucleotide Tertiary Structure Immune Modulation

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Solution Overview

Problem

Current treatments with cytokines and oligonucleotides for inflammatory and autoimmune diseases often face challenges such as significant side effects and immunogenicity, with a need for more effective methods to induce specific cytokine expression without unwanted activities.

Innovation Solution

Development of oligonucleotides that modulate the immune system by adopting specific tertiary structures, such as telomeric G-quadruplex tetramer or non-G-quadruplex dimer structures, to induce specific cytokine profiles like IFN-α, IFN-β, IFN-γ, IL-6, and IL-10, independent of their primary sequence, allowing for targeted immune modulation.

Engineering Contradictions & Design Principles

VSEngineering Contradiction Analysis

1Reliability

If conventional cytokine treatments are used to treat inflammatory and autoimmune diseases, then therapeutic effect is achieved, but significant side effects and immunogenicity occur

Engineering Contradiction:
Improvetherapeutic effectVSAvoidside effects and immunogenicity
Core Design Contradiction:
ReliabilityVSObject-affected harmful factors

Solution Approach 1:

The invention changes the fundamental parameter of treatment from protein-based cytokines to nucleic acid-based oligonucleotides with specific tertiary structures. This parameter change allows induction of endogenous cytokine production rather than direct administration of cytokines, thereby achieving therapeutic effects while avoiding the immunogenicity and side effects associated with exogenous cytokine treatments

Inventive Principle:
Principle #35Parameter changes

Solution Approach 2:

The oligonucleotides act as intermediaries that induce the host's own cells to produce cytokines endogenously. Instead of directly administering cytokines that cause immunogenicity, the oligonucleotides serve as mediators that trigger the body's natural cytokine production mechanisms, thereby achieving the desired immune modulation without the harmful effects of exogenous cytokine administration

Inventive Principle:
Principle #24Intermediary (Mediator)

2Adaptability or versatility

If oligonucleotides are designed to induce specific cytokine expression, then targeted immune modulation is achieved, but complexity in identifying effective sequences increases

Engineering Contradiction:
Improvetargeted immune modulationVSAvoidsequence identification complexity
Core Design Contradiction:
Adaptability or versatilityVSDevice complexity

Solution Approach 1:

The invention shifts the focus from primary sequence design to tertiary structure design. Instead of searching through vast sequence spaces to find effective oligonucleotides, the approach is to design oligonucleotides that fold into specific tertiary structures (G-quadruplexes, triple helices, etc.). This parameter change from sequence-centric to structure-centric design dramatically reduces the complexity of identifying effective oligonucleotides while maintaining targeted immune modulation capability

Inventive Principle:
Principle #35Parameter changes

Solution Approach 2:

The invention establishes that multiple different primary sequences can fold into the same functional tertiary structure and produce the same immunomodulatory effect. This universality means that once a functional tertiary structure is identified, many different sequence variants can be used, reducing the complexity of sequence identification while maintaining versatility in targeted immune modulation

Inventive Principle:
Principle #6Universality (Multi-functionality)

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

These oligonucleotides effectively stimulate cytokine production, offering a more targeted and potentially less toxic approach to treating inflammatory and autoimmune diseases by inducing specific cytokine profiles, thereby improving treatment efficacy while minimizing side effects.

Implementation Method 1

G-quartets arise from the association of four adjacent G-bases assembled into a cyclic conformation. These structures are stabilized by von Hoogsteen hydrogen bonding and by base stacking interactions

Methodology Applied
Scientific EffectHydrogen bonding:

Implementation Method 2

G-quartets arise from the association of four adjacent G-bases assembled into a cyclic conformation. These structures are stabilized by von Hoogsteen hydrogen bonding and by base stacking interactions

Methodology Applied
Scientific EffectBase stacking interactions:

Implementation Method 3

These oligonucleotides effectively stimulate cytokine production, offering a more targeted and potentially less toxic approach to treating inflammatory and autoimmune diseases by inducing specific cytokine profiles

Methodology Applied
Scientific EffectCytokine induction:

Data Source

PatentEP3165607B1Biologically active oligonucleotides capable of modulating the immune system
Publication Date: 2021.04.07 INDEX PHARMA
  • EP3165607B1 patent drawingFigure 1A~1C
  • EP3165607B1 patent drawingFigure 1D~1F
  • EP3165607B1 patent drawingFigure 1G~1H

AI summary

The present invention relates to methods of identifying oligonucleotides capable of modulating the immune system in a mammalian subject, comprising analysis of which tertiary structural type said oligonucleotide adopts, in phosphate-buffered saline solution. Further, the invention provides oligonucleotides identifiable by the methods of the invention and to their use in methods of treating diseases, such as inflammatory diseases, autoimmune diseases, infectious diseases, neurodegenerative diseases and cancer.