Methods and pharmaceutical compositions for the treatment of diseases associated with reduced CFTR function

US11458118B2 · kind B2 · utility

Assignees

• INSERM (INSTITUT NATIONAL DE LA SANTÉ ET DE LA RECHERCHE MÉDICALE) · FR
• Universite De Bretagne Occidentale · FR
• ETABLISSEMENT FRANCAIS DU SANG (EFS) · FR
• Centre Hospitalier Regional Et Universitaire De Brest · FR

Inventors

• Pascal Trouve · Brest, FR
• Claude Ferec · Plougastel-Daoulas, FR
• Mathieu Kerbiriou · Brest, FR
• Florentin Huguet · Brest, FR

Key dates

• Filing date: Apr 20, 2018
• Grant date: Oct 4, 2022
• Expiry date: May 17, 2038

Classification

• Technology area (CPC C): Chemistry; Metallurgy
• CPC primary: C12N2310/11
• WIPO field: Pharmaceuticals
• WIPO sector: Chemistry

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.