[4-[5-[2-chloro-4-(1H-pyrazol-4-yl)phenyl]-1,3,4-thiadiazol-2-yl]piperazin-2-yl]methanol

• 分子结构分类: 有机化合物 - 有机杂环化合物 - 二嗪烷类
• 英文名称: [4-[5-[2-chloro-4-(1H-pyrazol-4-yl)phenyl]-1,3,4-thiadiazol-2-yl]piperazin-2-yl]methanol
• 化学式: C₁₆H₁₇ClN₆OS
• 分子量: 376.9
• InChiKey: VJFXTTHIBMOYDP-UHFFFAOYSA-N

计算性质

• 辛醇/水分配系数(LogP)：
  1.8
• 重原子数：
  25
• 可旋转键数：
  4
• 环数：
  4.0
• sp3杂化的碳原子比例：
  0.31
• 拓扑面积：
  118
• 氢给体数：
  3
• 氢受体数：
  7

文献信息

• 1,4-DISUBSTITUTED PYRIDAZINE COMPOUNDS FOR TREATING HUNTINGTON'S DISEASE
  申请人：PTC Therapeutics, Inc.

  公开号：EP3848035A1

  公开（公告）日：2021-07-14

  The present description relates to compounds, forms, and pharmaceutical compositions thereof and methods of using such compounds, forms, or compositions thereof for treating or ameliorating Huntington's disease. In particular, the present description relates to substituted monocyclic heteroaryl compounds of Formula (I), forms and pharmaceutical compositions thereof and methods of using such compounds, forms, or compositions thereof for treating or ameliorating Huntington's disease.

  本说明涉及其化合物、形式和药物组合物，以及使用此类化合物、形式或组合物治疗或改善亨廷顿氏病的方法。 特别是，本说明书涉及式（I）的取代单环杂芳基化合物、其形式和药物组合物以及使用此类化合物、形式或其组合物治疗或改善亨廷顿氏病的方法。
• Methods for treating Huntington's disease
  申请人：PTC Therapeutics, Inc.

  公开号：US10874672B2

  公开（公告）日：2020-12-29

  The present description relates to compounds, forms, and pharmaceutical compositions thereof and methods of using such compounds, forms, or compositions thereof for treating or ameliorating Huntington's disease. In particular, the present description relates to substituted monocyclic heteroaryl compounds of Formula (I), forms and pharmaceutical compositions thereof and methods of using such compounds, forms, or compositions thereof for treating or ameliorating Huntington's disease.

  本说明涉及其化合物、形式和药物组合物，以及使用此类化合物、形式或组合物治疗或改善亨廷顿氏病的方法。 特别是，本说明书涉及式（I）的取代单环杂芳基化合物、其形式和药物组合物以及使用此类化合物、形式或其组合物治疗或改善亨廷顿氏病的方法。
• METHODS FOR TREATING HUNTINGTON'S DISEASE
  申请人：PTC Therapeutics, Inc.

  公开号：EP3386511B1

  公开（公告）日：2021-05-12
• METHODS FOR MODIFYING RNA SPLICING
  申请人：PTC THERAPEUTICS, INC.

  公开号：US20200370043A1

  公开（公告）日：2020-11-26

  Described herein is an intronic recognition element for splicing modifier (iREMS) that can be recognized by a small en molecule splicing modifier compound of Formula (I) provided herein or a form thereof, wherein W, X, A and B are as defined herein. In one aspect, methods for modifying RNA splicing to modulate the amount of a product of a gene, wherein a precursor RNA transcript transcribed from the gene that contains an intronic REMS is modified utilizing a splicing modifier compound of Formula (I), are described herein. In another aspect, methods for modifying RNA splicing to modulate the amount of an RNA transcript or protein product encoded by a gene, wherein a precursor RNA transcript transcribed from the gene is modified to comprise an intronic REMS utilizing a splicing modifier compound of Formula (I), are described herein.