Quinazoline Derivatives for Aberrant Splicing Suppression
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Solution Overview
Problem
There is a need for compounds that can suppress aberrant splicing, which contributes to the onset or progression of genetic diseases caused by aberrant splicing.
Innovation Solution
Development of a compound represented by Formula (I) or its pharmaceutically acceptable salts, which can suppress aberrant splicing by targeting specific molecular structures, including halogen atoms, C1-C6 alkyl groups, C3-C6 cycloalkyl groups, 4- to 10-membered heterocyclyl groups, and 5- to 12-membered aryl or heteroaryl groups, with specific substituents.
Engineering Contradictions & Design Principles
Engineering Contradiction Analysis
1Reliability
If a compound is designed to suppress aberrant splicing by targeting specific molecular structures, then the therapeutic effectiveness for genetic diseases is improved, but the complexity of the compound structure increases
Solution Approach 1:
The patent systematically varies chemical parameters including halogen atoms (F, Cl, Br, I), alkyl group sizes (C1-C6), cycloalkyl group sizes (C3-C6), heterocyclyl group sizes (4- to 10-membered), and aryl/heteroaryl group sizes (5- to 12-membered) to optimize splicing suppression activity while managing structural complexity
Solution Approach 2:
The compound structure incorporates specific functional groups at defined positions (R1-R6 substituents on the quinazoline core) to target specific molecular structures involved in aberrant splicing, with each substituent position contributing differently to the overall therapeutic effectiveness
2Reliability
If multiple substituent groups are incorporated into the compound to enhance splicing suppression, then the activity against aberrant splicing is improved, but the ease of manufacture decreases
Solution Approach 1:
The compound is divided into a core quinazoline structure (providing splicing suppression activity) and multiple substituent groups (R1-R6) that can be independently optimized, allowing systematic structure-activity relationship studies and facilitating manufacturing by modular synthesis approaches
Data Source
Figure 1
AI summary
An aspect of the present disclosure provides a compound capable of suppressing aberrant splicing that contributes to the onset or progression of a disease, or a pharmaceutically acceptable salt thereof. An aspect of the present disclosure relates to a compound represented by Formula (I) below or a pharmaceutically acceptable salt thereof.