Compounds for the treatment of cystic fibrosis

WO2026060296A3 Publication Date: 2026-04-23AUGELLI SZAFRAN CORINNE E +3
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Patent Information

Application Number
PCT/US2025/046238
Authority / Receiving Office
WO · WO
Patent Type
Applications
Current Assignee / Owner
Priority Date
2024-09-13
Filing Date
2025-09-12
Publication Date
2026-04-23

AI Technical Summary

Technical Problem

Current pharmacological strategies for treating genetic diseases caused by premature termination codons, such as cystic fibrosis, are not optimal in terms of efficacy and safety, and there is a need for compounds that can effectively stimulate read-through of these codons.

Method used

Development of substituted piperidinedione compounds and pharmaceutical compositions that modulate the read-through of premature termination codons, including specific structures and their pharmaceutically acceptable salts, which can be administered to subjects to treat disorders associated with these codons.

Benefits of technology

The compounds effectively promote the read-through of premature termination codons, potentially restoring functional protein and treating disorders like cystic fibrosis, Duchenne muscular dystrophy, and other genetic conditions.

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Abstract

The present disclosure is concerned with piperidinedione compounds, pharmaceutical compositions comprising the compounds, and methods of treating disorders associated with the presence of a premature termination codon such as, for example, cystic fibrosis, Duchenne muscular dystrophy, aniridia, Becker muscular dystrophy, spinal muscular atrophy, Hurler syndrome, hemophilia, epidermolysis bullosa (e.g., dystrophic (DEB) form, junctional (JEB) form). Usher syndrome, and cancer, using the compounds. This abstract is intended as a scanning tool for purposes of searching in the particular art and is not intended to be limiting of the present invention.
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