Alkyl Ether Derivative Binding Agent for Emopamil Protein
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Solution Overview
Problem
Current compounds do not effectively target Emopamil Binding Protein (EBP) for therapeutic intervention in male EBP disorders associated with X-linked dominant Conradi-Hunermann chondrodysplasia punctata, Smith-Lemli-Opitz Syndrome, lathosterolosis, or desmosterolosis, lacking high affinity and clear pharmacological action.
Innovation Solution
An alkyl ether derivative represented by a specific formula, or its salt, exhibits high affinity for EBP, serving as a binding agent for preventing and treating these disorders, with applications in both therapeutic and research contexts.
Engineering Contradictions & Design Principles
Engineering Contradiction Analysis
1Reliability
If existing compounds are used as EBP ligands, then they can bind to EBP, but they lack high affinity and clear pharmacological action
Solution Approach 1:
The patent applies parameter changes by modifying the chemical structure of emopamil through systematic variations in the alkyl ether derivative formula (Formula 1), changing substituents R1-R6, ring sizes m and n, and molecular weight parameters to optimize binding affinity while maintaining selectivity for EBP over other targets
Solution Approach 2:
The patent applies local quality by introducing specific functional groups at particular positions in the molecular structure (R1-R6 substituents), where each position's chemical properties are locally optimized to enhance interaction with specific amino acid residues in the EBP binding pocket, thereby improving both affinity and pharmacological clarity
2Reliability
If compounds with high affinity for EBP are developed, then therapeutic intervention becomes possible, but the pharmacological action remains unclear due to cross-binding with calcium channel and sigma-1 receptor
Solution Approach 1:
The patent applies local quality by designing specific substituent patterns at key positions (particularly R2, R3, and R4) that create a unique molecular recognition profile, allowing the compound to fit the EBP binding pocket geometry and electrostatic environment while avoiding complementary interactions with calcium channels and sigma-1 receptors
Solution Approach 2:
The patent applies inversion by reversing the conventional approach: instead of starting from known calcium channel or sigma-1 receptor ligands and trying to modify them, the invention starts from emopamil's EBP binding capability and systematically modifies it to enhance EBP selectivity, effectively inverting the target prioritization hierarchy
Data Source
Figure 1~2
AI summary
Required are: a compound having high affinity for an emopamil binding protein; and a method for activating or inhibiting the emopamil binding protein, using such a compound. According to the present invention, provided is an emopamil binding protein binding agent comprising an alkyl ether derivative represented by the following formula [1] or a salt thereof: wherein R1 and R2, which are the same or different, each represent a hydrogen atom, a halogen atom, an optionally substituted C1-6 alkyl group, an optionally substituted aryl group, etc.; R3 represents an optionally protected hydroxyl group, etc.; and m and n, which are the same or different, each represent an integer of 1 to 6.