4-amido-pyrimidine Modulators for ABC Transporter Activity
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Solution Overview
Problem
Current treatments for ABC transporter-mediated diseases, such as Cystic Fibrosis, lack effective modulators to address defective protein trafficking and activity, leading to impaired ion and fluid transport, and there is a need for compounds that can modulate ABC transporter activity to treat a range of diseases beyond CFTR-related conditions.
Innovation Solution
Development of compounds with a specific formula that modulate ABC transporter activity, including CFTR, by interacting with binding domains to increase or decrease activity, thereby treating various diseases associated with defective protein trafficking and ion transport.
Engineering Contradictions & Design Principles
Engineering Contradiction Analysis
1Reliability
If current treatments for ABC transporter-mediated diseases are used, then existing therapeutic options are available, but effective modulators to address defective protein trafficking and activity are lacking
Solution Approach 1:
The patent develops novel compounds with specific chemical structures (formula I and II) that represent parameter changes in molecular design to create effective modulators of ABC transporter activity, addressing the lack of available treatments for diseases like Cystic Fibrosis
2Reliability
If compounds are developed to modulate ABC transporter activity, then diseases like Cystic Fibrosis can be treated, but the complexity of identifying effective modulators increases
Solution Approach 1:
The patent presents segmented approaches to compound development through multiple formula variations (formula I with specific substituents Ra, Rb, Rc, Rd and formula II), breaking down the complex task of modulator development into manageable structural categories and subclasses
Solution Approach 2:
The developed compounds are designed to modulate ABC transporter activity broadly, with potential application to multiple diseases beyond Cystic Fibrosis including other protein folding disorders and secretory diseases, providing universal therapeutic value
3Productivity
If modulators are used to enhance CFTR activity, then ion and fluid transport is improved, but the underlying defects in protein trafficking must be addressed
Solution Approach 1:
The patent develops compound modulators that act as intermediary substances to enhance CFTR activity and correct protein trafficking defects, serving as a bridge between the defective transporter and functional recovery, thereby improving ion and fluid transport while addressing underlying trafficking issues
Data Source
AI summary
4-amido-pyrimidine compounds, derivatives and compositions thereof, and synthetic methods described herein are useful for modulating ATP-Binding Cassette (“ABC”) transporters or fragments thereof, including Cystic Fibrosis Transmembrane Conductance Regulator (“CFTR”). The present invention also relates to methods of treating ABC transporter mediated diseases using such modulators.