Methods and pharmaceutical compositions for the treatment of diseases associated with reduced CFTR function
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United States of America Patent
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Oct 4, 2022
Grant Date -
Apr 23, 2020
app pub date -
Apr 20, 2018
filing date -
Apr 21, 2017
priority date (Note) -
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Abstract
Activating transcription factor 6 (ATF6) is involved in cystic fibrosis transmembrane conductance regulator (CFTR) repression and understanding this inhibitory mechanism is of great interest to develop a therapeutic approach based on UPR regulation. Using site-1 protease (S1P) inhibitor (e.g. PF-429242) the inventors showed that both membrane localization and function of F508del-CFTR are partially restored. Accordingly, the present invention relates to a method of treating a disease associated with reduced CFTR function in a subject in need thereof comprising administering to the subject a therapeutically effective amount of a S1P inhibitor.
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