CFTR Modulator Compositions for Broader Mutation Response
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Solution Overview
Problem
Current treatments for cystic fibrosis, such as lumacaftor and tezacaftor, do not effectively address all CFTR mutations and can cause side effects, necessitating the development of alternative therapeutic strategies to manage CFTR-related diseases and respiratory distress.
Innovation Solution
Development of CFTR modulators, including compounds like 3-(2,3-dichlorophenyl)-6-(tert-pentyl)-7H-[1,2,4]triazolo[3,4-b][1,3,4]thiadiazine and N-(2-(cyclobutanecarboxamido)benzo[d]thiazol-6-yl)-1,3-dimethyl-1H-pyrazole-4-carboxamide, which are administered to increase CFTR activity and reduce respiratory distress.
Engineering Contradictions & Design Principles
Engineering Contradiction Analysis
1Reliability
If current CFTR modulators (lumacaftor, tezacaftor) are used to treat CFTR mutations, then some processing abnormalities are overcome, but side effects occur and not all mutations are effectively addressed
Solution Approach 1:
The patent applies parameter changes by developing new chemical compounds with modified molecular structures (different chemical formulas and properties) to achieve improved therapeutic efficacy and reduced side effects compared to existing CFTR modulators. The new compounds represent parameter changes in chemical composition and structure to overcome limitations of current treatments.
2Reliability
If current CFTR modulator therapies are administered, then some CFTR processing abnormalities are overcome, but the ability to tolerate long term treatment is limited
Solution Approach 1:
The patent employs parameter changes by creating new chemical entities with optimized properties that aim to improve long-term treatment tolerance. The modified chemical parameters of the new compounds are designed to maintain therapeutic benefits while reducing adverse effects that limit long-term use.
Solution Approach 2:
The patent introduces new CFTR modulator compounds as intermediary substances that mediate between the therapeutic goal and the biological system. These intermediary compounds are designed to achieve the desired therapeutic effect with improved safety and tolerability profiles for long-term treatment.
3Adaptability or versatility
If additional therapeutic strategies are developed to address non-responders, then coverage of more CFTR mutations is achieved, but treatment complexity increases
Solution Approach 1:
The patent applies universality by developing new CFTR modulator compounds that aim to address multiple CFTR mutation types with a single therapeutic agent. The new compounds are designed with broad applicability to cover various mutations, reducing the need for multiple specialized treatments.
Solution Approach 2:
The patent uses parameter changes in chemical structure to create compounds with enhanced versatility in addressing different CFTR mutations. By modifying chemical parameters, the new compounds achieve broader mutation coverage while maintaining manageable treatment complexity.
Data Source
AI summary
This disclosure relates to compounds that are cystic fibrosis transmembrane conductance regulator (CFTR) modulators and pharmaceutical compositions containing the same. In certain embodiments, this disclosure relates to methods of managing a CFTR related disease or condition or respiratory distress comprising administering an effective amount of a CFTR modulator disclosed herein to a subject in need thereof.