CFTR Modulator Compounds Enhance Anion Secretion
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Solution Overview
Problem
Current treatments for cystic fibrosis and related conditions, such as chronic obstructive pulmonary disease and dry eye disease, lack effective modulators for the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein, which are essential for managing anion transport and fluid balance, leading to inadequate hydration and mucus clearance.
Innovation Solution
Development of novel compounds, represented by Formula (I), that act as CFTR modulators to enhance anion secretion and fluid transport, potentially improving symptoms in cystic fibrosis and other diseases by correcting defective protein trafficking and function.
Engineering Contradictions & Design Principles
Engineering Contradiction Analysis
1Reliability
If conventional treatments are used for cystic fibrosis, then patient management is maintained, but anion transport and fluid balance are insufficient leading to inadequate hydration and mucus clearance
Solution Approach 1:
The patent applies parameter changes by developing novel chemical compounds with specific molecular structures (Formula I) that bind to and modulate CFTR protein activity. These compounds change the functional parameters of CFTR, enhancing anion secretion and fluid transport capabilities to overcome the insufficiency in cystic fibrosis patients
Solution Approach 2:
The patent uses small molecule compounds as intermediaries that mediate between the defective CFTR protein and the desired physiological outcome. These compounds act as pharmacological chaperones or modulators that interact with CFTR to restore its proper folding, trafficking, and function, thereby improving anion transport and fluid balance
2Productivity
If CFTR modulators are developed to enhance anion secretion, then hydration and mucociliary clearance improve, but the complexity of treating multiple CFTR-related conditions increases
Solution Approach 1:
The patent demonstrates universality by developing CFTR modulator compounds that can treat multiple different conditions caused by CFTR dysfunction. The same compound class addresses cystic fibrosis, chronic obstructive pulmonary disease, and dry eye disease through a common mechanism of enhancing CFTR-mediated anion secretion and fluid transport, thereby simplifying treatment approaches across different diseases
Data Source
AI summary
The present invention relates to compounds and their use in the treatment of cystic fibrosis, methods for their production, pharmaceutical compositions comprising the same, and methods of treating cystic fibrosis by administering a compound of the invention.