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US 2026/0078136 A1Published application
Compounds, compositions, and methods for modulating cftr
AssigneeProteostasis Therapeutics, Inc.
InventorsBenito Munoz, Daniel Parks, Cecilia M. Bastos
Life of this patent
Estimating the term…
Abstract
The present disclosure is directed to disclosed compounds that modulate e.g., address underlying defects in cellular processing of CFTR activity.
Claims27
A compound selected from N-(2,4-di-tert-butyl-5-hydroxyphenyl)-4-oxo-1,4-dihydroquinoline-3-carboxamide and a pharmaceutically acceptable salt thereof, wherein at least one carbon atom is replaced by silicon.
A compound represented by Formula I:
- or a pharmaceutically acceptable salt thereof, wherein:
- X1 is selected from the group consisting of —Si(R3)3, —C(CY3)3, hydrogen, halogen, C1-3alkyl optionally substituted by one or more halogens, C3-6cycloalkyl, and 4-6 membered saturated monocyclic heterocyclyl;
- wherein C3-6cycloalkyl and 4-6 membered saturated monocyclic heterocyclyl may be optionally be substituted by one, two, three or more substituents each independently selected for each occurrence from R11;
- X2 is selected from the group consisting of —Si(R3)3 and —C(CY3)3;
- wherein at least one of X1 or X2 is —Si(R3)3;
- R3 is independently selected for each occurrence from the group consisting of hydroxyl, C1-4alkyl, C1-4alkoxy, and phenyl, wherein C1-4alkyl, C1-4alkoxy, and phenyl may optionally be substituted by one, two, three or more deuterium atoms;
- or two R3 groups together with the silicon to which they are attached form a 4-6 membered saturated cyclosilane;
- Y is independently selected for each occurrence from the group consisting of hydrogen and deuterium;
- and
- R11 is independently selected for each occurrence from the group consisting of hydrogen, halogen, hydroxyl, cyano, and C1-6alkyl;
- wherein C1-6alkyl may be optionally substituted by one or more substituents each independently selected for each occurrence from the group consisting of halogen, hydroxyl, and C1-6alkoxy.
8 more elements
A compound selected from the group consisting of:
- and a pharmaceutically acceptable salt thereof;
- wherein any atom not designated as deuterium is present at its natural isotopic abundance.
Dependencies are inferred from the claim text.