Modulating Ciliogenesis via Chromatin Complex Binding

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Solution Overview

Problem

Chronic obstructive pulmonary disease (COPD) is characterized by inefficient mucociliary clearance due to loss of ciliated cells, leading to severe symptoms and high mortality, as the abnormal bronchial epithelium fails to clear airway mucus, bacteria, and debris effectively.

Innovation Solution

Compounds that bind or interact with chromatin remodeling complexes, such as Polycomb Repressor Complex 1 (PRC1) and Trithorax group-MLL (TrxG-MLL), are used to modulate ciliogenesis, acting as an 'on/off' switch for ciliogenesis in the human bronchial epithelium, thereby treating or preventing aberrant or defective ciliogenesis-related diseases.

Engineering Contradictions & Design Principles

VSEngineering Contradiction Analysis

1Reliability

If compounds bind to chromatin remodeling complexes (PRC1/TrxG-MLL) to modulate ciliogenesis, then ciliogenesis is restored/enhanced in COPD patients, but the complexity of the treatment mechanism increases

Engineering Contradiction:
Improveciliogenesis restorationVSAvoidtreatment mechanism complexity
Core Design Contradiction:
ReliabilityVSDevice complexity

Solution Approach 1:

The patent uses small molecule compounds as intermediaries to modulate the interaction between R2R1 protein and chromatin remodeling complexes (PRC1/TrxG-MLL). These compounds act as mediators that can enhance or inhibit ciliogenesis by affecting the R2R1-chromatin complex interaction, thereby providing a controllable way to restore ciliogenesis in COPD patients without directly manipulating the complex protein-protein interactions

Inventive Principle:
Principle #24Intermediary (Mediator)

Solution Approach 2:

The patent employs compounds that can change the functional state of chromatin remodeling complexes by binding to them or their associated proteins (R2R1). By modifying parameters such as chromatin accessibility, histone modification states, or protein interaction dynamics, the compounds enable controlled modulation of ciliogenesis, transforming a complex biological process into a regulatable therapeutic effect

Inventive Principle:
Principle #35Parameter changes

2Ease of operation

If R2R1 protein interaction with PRC1/TrxG-MLL complexes is modulated to control ciliogenesis, then ciliogenesis can be switched on/off, but the difficulty of detecting and measuring the mechanism increases

Engineering Contradiction:
Improveciliogenesis controlVSAvoidmechanism detection difficulty
Core Design Contradiction:
Ease of operationVSDifficulty of detecting and measuring

Solution Approach 1:

The patent utilizes compounds that can induce detectable changes in the system, potentially through fluorescent tagging or colorimetric assays associated with chromatin remodeling activity. By linking ciliogenesis modulation to observable physical changes (such as fluorescence intensity, color change, or other detectable signals), the mechanism becomes measurable and monitorable, bridging the gap between molecular interaction and detectable output

Inventive Principle:
Principle #32Color changes

3Adaptability or versatility

If compounds target specific chromatin binding/remodeling complexes to treat COPD, then treatment specificity is improved, but the precision of targeting required increases

Engineering Contradiction:
Improvetreatment specificityVSAvoidtargeting precision
Core Design Contradiction:
Adaptability or versatilityVSManufacturing precision

Solution Approach 1:

The patent employs compounds with specific molecular structures designed to bind preferentially to particular chromatin remodeling complexes (PRC1 or TrxG-MLL) or their interaction interfaces with R2R1. By optimizing local molecular properties such as binding affinity, specificity constants, and structural complementarity, the compounds achieve high targeting precision for specific complexes while maintaining versatility in treating different COPD cases through selective modulation

Inventive Principle:
Principle #3Local quality

Data Source

PatentEP3377106B1Modulation of ciliogenesis
Publication Date: 2023.05.24 ACADEMISCH ZIEKENHUIS LEIDEN (H O D N LUMC)
  • EP3377106B1 patent drawingFigure 1
  • EP3377106B1 patent drawingFigure 2
  • EP3377106B1 patent drawingFigure 3

AI summary

The disclosure is based on the finding that compounds capable of binding to (or interacting with) chromatin binding/remodelling complexes (for example Polycomb group PRC1 and Trithorax group MLL) and/or modulation of the same can be used to modulate (for example switch on/off) ciliogenesis as may occur, for example, in the human pulmonary bronchial epithelium. Provided are compounds, compositions, methods and medicaments which may be used to treat and/or prevent diseases and/or conditions associated with aberrant or defective ciliogenesis.