BHBA Compounds for RARβ2 Agonist Activity in Nerve Injury

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Solution Overview

Problem

Current treatments for nerve injuries, including spinal cord injuries, lack effective solutions for promoting axonal outgrowth and functional recovery due to the presence of growth inhibiting molecules, insufficiency of growth-promoting factors, and lack of appropriate growth programs in damaged neurons.

Innovation Solution

Development of bicycloheteroaryl-heteroaryl-benzoic acid compounds (BHBA compounds) that selectively activate retinoic acid receptor beta (RARβ2), promoting neurite development, outgrowth, and regeneration by mimicking the retinoid signaling pathway to overcome growth inhibitions and induce appropriate growth programs in neurons.

Engineering Contradictions & Design Principles

VSEngineering Contradiction Analysis

1Reliability

If conventional treatments are used for nerve injuries, then current standard therapies are applied, but they fail to promote axonal outgrowth and functional recovery due to growth inhibiting molecules and insufficiency of growth-promoting factors

Engineering Contradiction:
Improveeffectiveness of nerve injury treatmentVSAvoidaxonal outgrowth rate
Core Design Contradiction:
ReliabilityVSProductivity

Solution Approach 1:

The patent changes the chemical parameters by developing BHBA compounds with specific molecular structures (bicycloheteroaryl-heteroaryl-benzoic acid framework) that selectively activate RARβ2 receptors. This parameter change in molecular structure enables the compounds to overcome growth inhibitions and promote axonal outgrowth where conventional treatments fail

Inventive Principle:
Principle #35Parameter changes

Solution Approach 2:

The BHBA compounds act as intermediary substances that mediate between the administered treatment and the RARβ2 receptors in damaged neurons. These compounds selectively bind to and activate RARβ2, serving as a bridge to restore the retinoid signaling pathway and induce appropriate growth programs in injured neurons

Inventive Principle:
Principle #24Intermediary (Mediator)

2Productivity

If growth-promoting factors are increased to promote neurite outgrowth, then axonal regeneration may be enhanced, but the presence of growth inhibiting molecules counteracts this effect

Engineering Contradiction:
Improveneurite outgrowth rateVSAvoidgrowth inhibiting molecules
Core Design Contradiction:
ProductivityVSObject-affected harmful factors

Solution Approach 1:

The patent converts the harmful effect of growth inhibiting molecules into a beneficial outcome by using BHBA compounds that selectively activate RARβ2. The compounds overcome the inhibitory environment by triggering a specific signaling pathway (retinoid signaling through RARβ2) that promotes axonal outgrowth despite the presence of growth inhibiting molecules like Nogo-A, MAG, and Omgp

Inventive Principle:
Principle #22Blessing in disguise (Convert harm into benefit)

3Adaptability or versatility

If RARβ2 is selectively activated to induce appropriate growth programs, then neurite development is promoted, but existing treatments lack the specificity to target RARβ2 without affecting other RAR subtypes

Engineering Contradiction:
Improveselectivity of RAR activationVSAvoidmolecular structure complexity
Core Design Contradiction:
Adaptability or versatilityVSDevice complexity

Solution Approach 1:

The BHBA compounds exhibit local quality in their receptor interaction by selectively binding to and activating RARβ2 while having minimal effect on other RAR subtypes (RARα, RARγ). This selective activation is achieved through specific molecular features of the BHBA structure that match the binding pocket of RARβ2, enabling targeted therapy without broad RAR activation

Inventive Principle:
Principle #3Local quality

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

BHBA compounds effectively initiate axonal outgrowth and functional recovery in models of nerve injury by selectively activating RARβ2, addressing the limitations of existing treatments by enhancing neurite development and regeneration.

Implementation Method 1

the RARβ2 subtype is specifically involved in this process... Activation of RARβ2 by retinoids results in neurite outgrowth of cultured embryonic dorsal root ganglia (DRG), spinal cord, and adult DRG

Methodology Applied
Scientific EffectRetinoid signaling pathway:

Data Source

PatentEP3233842B1Bicycloheteroaryl-heteroaryl-benzoic acid compounds as retinoic acid receptor beta (RARB) agonists
Publication Date: 2025.03.26 KINGS COLLEGE LONDON
  • EP3233842B1 patent drawingFigure 1
  • EP3233842B1 patent drawingFigure 2(A)~2(B)
  • EP3233842B1 patent drawing

AI summary

The present invention pertains generally to the field of therapeutic compounds, and more specifically to certain bicycloheteroaryl-heteroaryl-benzoic acid compounds of the following formula (for convenience, collectively referred to herein as "BHBA compounds"), which, inter alia, are (selective) retinoic acid receptor beta (RARβ) (e.g., RARβ2) agonists. The present invention also pertains to pharmaceutical compositions comprising such compounds, and the use of such compounds and compositions, both in vitro and in vivo, to (selectively) activate RARβ (e.g., RARβ2), to cause or promote neurite development, neurite outgrowth, and/or neurite regeneration, and in the treatment of diseases and conditions that are mediated by RARβ (e.g., RARβ2), that are ameliorated by the activation of RARβ (e.g., RARβ2), etc., including, e.g., neurological injuries such as spinal cord injuries.