Compounds, compositions, and methods of use thereof

By developing drugs containing compounds of formula (I) or (II), the problem of CFTR protein dysfunction in cystic fibrosis has been solved, and effective treatment and symptom improvement of the disease has been achieved.

CN119677730APending Publication Date: 2025-03-21SIONNA THERAPEUTICS INC
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Patent Information

Application Number
CN202380058226.4
Authority / Receiving Office
CN · China
Patent Type
Applications(China)
Current Assignee / Owner
Priority Date
2022-06-10
Filing Date
2023-06-09
Publication Date
2025-03-21

AI Technical Summary

Technical Problem

The deletion or dysfunction of CFTR protein in patients with cystic fibrosis leads to exocrine dysfunction and multisystem diseases, and existing treatments cannot effectively cure this disease.

Method used

A drug comprising a compound of formula (I) or (II) is developed to treat cystic fibrosis and other CFTR-mediated diseases by its pharmaceutically acceptable salts and methods of use.

Benefits of technology

This compound can effectively regulate the function of CFTR channels, improve clinical symptoms in patients, reduce the severity of the disease, and provide new methods for treating CFTR-related diseases.

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Abstract

The present invention relates to heterocyclic compounds, pharmaceutically acceptable salts thereof, and pharmaceutical formulations thereof. Also described herein are compositions and the use of such compounds in methods of treating diseases and disorders mediated by deficient CFTR activity, in particular cystic fibrosis.
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