CFTR Modulator Composition for Restoring Anion Transport in Cystic Fibrosis
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Solution Overview
Problem
Current treatments for cystic fibrosis (CF) are inadequate, as mutations in the CFTR protein lead to impaired anion and fluid transport, resulting in mucus accumulation, microbial infections, and other severe health issues, with no cure available.
Innovation Solution
Development of Compound I, a CFTR modulator, which can be administered in pharmaceutical compositions to correct CFTR protein trafficking and enhance channel activity, potentially alleviating the symptoms of CF.
Engineering Contradictions & Design Principles
Engineering Contradiction Analysis
1Duration of action of stationary object
If current treatments for cystic fibrosis are used, then patient survival is extended, but the underlying CFTR protein dysfunction remains untreated and disease progression continues
Solution Approach 1:
The patent employs CFTR modulators as intermediary substances that bridge the gap between the defective CFTR protein and functional anion transport. These modulators act as mediators that bind to the mutant CFTR protein and restore its function, enabling it to perform its physiological role in anion and fluid transport across epithelial cells.
Solution Approach 2:
The invention utilizes parameter changes in the CFTR protein's physical and functional properties through pharmacological intervention. By administering modulators that alter the conformational state, stability, and gating properties of the CFTR protein, the patent transforms the protein's functional parameters from defective to near-normal, thereby restoring physiological anion transport.
2Reliability
If CFTR modulators are administered to correct protein trafficking, then anion and fluid transport is improved, but the complexity of treatment regimens increases
Solution Approach 1:
The patent combines multiple CFTR modulators with different mechanisms of action into a single therapeutic regimen. By merging correctors that address trafficking defects with potentiators that enhance channel function, the treatment achieves comprehensive restoration of CFTR activity through a coordinated multi-component approach that targets multiple aspects of protein dysfunction simultaneously.
3Object-affected harmful factors
If mutations in CFTR are left untreated, then disease progression continues leading to mucus accumulation and infections, but aggressive interventions may cause adverse effects
Solution Approach 1:
The patent converts the harmful effect of mutant CFTR protein misfolding into a beneficial outcome by utilizing the fact that the mutant protein retains some residual function. By applying modulators that stabilize and enhance this residual function, the treatment transforms the previously harmful defective protein into a functional therapeutic target, achieving disease modification without requiring protein replacement or gene therapy.
Data Source
AI summary
Compound I of the formulaand/or pharmaceutically acceptable salt(s) of Compound I comprised in a pharmaceutical composition and methods of using the same to treat cystic fibrosis.