Compositions and methods for treating huntington's disease and htt proteinopathies

• Main Author: KEEFE DENNIS
• Format: Patent
• Language: Chinese; English
• Published: 21.02.2025
• Subjects: HUMAN NECESSITIES; HYGIENE; MEDICAL OR VETERINARY SCIENCE; PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES; SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS

The present disclosure provides compounds, compositions and/or methods for treating, preventing, inhibiting, ameliorating, or delaying the onset of Huntington's disease and/or HTT proteinopathy in a subject. The methods may include administering to the subject an effective amount of a peptidomimetic compound, such as (R)-2-amino-N-((S)-1-(((S)-5-amino-1-(3-benzyl-1, 2, 4-oxadiazol-5-yl) pentyl) amino)-3-(4-hydroxy-2, 3, 4-triazole-3-yl)-3-(4-hydroxyl-2, 3, 4-triazole-3-yl)-3-(4-hydroxyl-2, 3, 4-triazole-3-yl)-3-(4-hydroxyl-2, 3, 4-triazole-3-yl)-3-(4- The present invention relates to a pharmaceutical composition, in particular, a pharmaceutically acceptable salt, stereoisomer, tautomer, hydrate and/or solvate thereof, or a pharmaceutically acceptable salt, stereoisomer, tautomer, hydrate and/or solvate thereof. 本公开提供了用于治疗、预防、抑制、改善受试者的亨廷顿病和/或HTT蛋白病或延迟其发作的化合物、组合物和/或方法。所述方法可以包括单独地或与一种或多种其他治疗剂组合地向所述受试者施用有效量的肽模拟物化合物，诸如(R)-2-氨基-N-((S)-1-(((S)-5-氨基-1-(3-苄基-1,2,4-噁二唑-5-基)戊基)氨基)-3-(4-羟基-2,6-二甲基苯基)-1-氧代丙烷-2-基)-5-胍基戊酰胺，