Composition for use in the treatment of ApoL1-associated disease
Inventors
Skorecki, Karl • BAVLI-KERTSELLI, Ira • SHEMER, Tali • Kruzel-Davila, Etty • TABACHNIKOV, Orly
Assignees
Technion Research and Development Foundation Ltd • Rambam Med Tech Ltd • Vertex Pharmaceuticals Inc
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Publication Number
US-12629376-B2
Publication Date
2026-05-19
Expiration Date
Abstract
The invention in some embodiments thereof, relates to the field of compositions and/or pharmaceutical compositions comprising one or more biologically active compound, and is directed to methods of using same such as for treating an ApoL1 related disease or a disorder in a subject.
Core Innovation
The disclosed invention concerns quinazolinedione and related compound families, including compounds represented by Formula 1D, Formula 4.1, Formula 6D/6E, and pharmaceutical compositions containing them. The subject matter describes administering a compound or a pharmaceutically acceptable salt to a subject in need thereof to treat ApoL1-associated kidney disease. The compound definitions encompass specified substituent choices, structural features, and specific depicted compounds.
The provided content describes reducing ApoL1-associated cell toxicity, including toxicity associated with ApoL1 G1 and G2 variants, while maintaining substantial inactivity toward wild-type ApoL1 cells. Findings report dose-dependent improvement in cell viability in mutant-expressing cells and potential antiproliferative activity based on reduced viability in immortalized cells. The problem identified in the background section is not explicitly described in the provided patent content.
Claims Coverage
The provided claims identify three inventive features across independent claims 1, 4, and 5.
Treatment using compounds of Formula 1D or Formula 4.1
Administering a therapeutically effective amount of a compound or pharmaceutically acceptable salt represented by Formula 1D or Formula 4.1 to treat ApoL1-associated kidney disease, subject to the stated substituent and structural definitions.
Treatment using an identified compound or salt
Administering a therapeutically effective amount of a compound or pharmaceutically acceptable salt to a subject in need thereof for treating ApoL1-associated kidney disease, wherein the supplied claim text ends before identifying the compound.
Compound or pharmaceutically acceptable salt
A depicted compound or a pharmaceutically acceptable salt thereof, wherein the specific structure is not visible in the supplied claim text.
The independent claims cover methods of treating ApoL1-associated kidney disease using compounds of Formula 1D or Formula 4.1 or an unspecified compound or salt, as well as a depicted compound or its pharmaceutically acceptable salt.
Stated Advantages
The supplied content reports dose-dependent improvement in cell viability in cells expressing ApoL1 G1 and G2 variants, with substantial inactivity toward wild-type ApoL1 cells.
Documented Applications
Treatment of ApoL1-associated kidney disease by administering a claimed compound or pharmaceutically acceptable salt to a subject in need thereof.
Reducing ApoL1-associated cell toxicity, including toxicity associated with ApoL1 G1 and G2 variants.
A pharmaceutical composition comprising the compound of claim 5 or its pharmaceutically acceptable salt and a pharmaceutically acceptable carrier.
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