CFTR Modulators Correct Protein Folding and Enhance Anion Secretion
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Solution Overview
Problem
Current treatments for ABC transporter-mediated diseases, particularly those affecting CFTR, are inadequate in effectively modulating protein activity to address defects in ion and fluid transport, leading to conditions like cystic fibrosis and other secretory disorders.
Innovation Solution
Development of compounds that modulate ABC transporter activity, specifically targeting CFTR, to enhance anion secretion and correct protein folding defects, thereby improving ion and fluid transport across epithelial membranes.
Engineering Contradictions & Design Principles
Engineering Contradiction Analysis
1Reliability
If current treatments for ABC transporter-mediated diseases are used, then some therapeutic effect is achieved, but they are inadequate in effectively modulating protein activity to address defects in ion and fluid transport
Solution Approach 1:
The patent develops novel compounds with modified chemical structures (formula I with various R groups and aromatic rings) to change the pharmacological parameters of CFTR modulation. These compounds differ from existing treatments in their molecular structure, allowing them to effectively modulate CFTR activity and address defects in ion and fluid transport that current treatments cannot adequately address
Solution Approach 2:
The patent creates new compounds that replicate and enhance the desired therapeutic effect of existing CFTR modulators while overcoming their limitations. By designing compounds with specific structural features (substituents, ring systems, linkers), the invention produces improved versions that more effectively correct protein folding defects and enhance anion secretion
2Productivity
If compounds are developed to enhance anion secretion and correct protein folding defects, then ion and fluid transport is improved, but the complexity of treating multiple disease manifestations increases
Solution Approach 1:
The patent describes compounds that perform multiple functions simultaneously: they correct protein folding defects, enhance anion secretion, and improve ion and fluid transport. This multi-functionality allows a single compound to address multiple disease manifestations of cystic fibrosis and other ABC transporter-mediated diseases, reducing the need for multiple separate treatments
Data Source
AI summary
The present invention relates to modulators of ATP-Binding Cassette (“ABC”) transporters or fragments thereof, including Cystic Fibrosis Transmembrane Conductance Regulator, compositions thereof, and methods therewith. The present invention also relates to methods of treating ABC transporter mediated diseases using such modulators.