5-(3,5-二氯苯氧基)-N-4-吡啶基 | 685830-90-4

• 分子结构分类: 有机化合物 - 有机氧化合物
• 中文名称: 5-(3,5-二氯苯氧基)-N-4-吡啶基
• 英文名称: 5-(3,5-dichlorophenoxy)-N-(pyridin-4-yl)furan-2-carboxamide
• 英文别名: 5-(3,5-dichlorophenoxy)-N-pyridin-4-ylfuran-2-carboxamide
• CAS: 685830-90-4
• 化学式: C₁₆H₁₀Cl₂N₂O₃
• 分子量: 349.2
• InChiKey: FRDJJGSXDLUKPF-UHFFFAOYSA-N

计算性质

• 辛醇/水分配系数(LogP)：
  4.2
• 重原子数：
  23
• 可旋转键数：
  4
• 环数：
  3.0
• sp3杂化的碳原子比例：
  0.0
• 拓扑面积：
  64.4
• 氢给体数：
  1
• 氢受体数：
  4

文献信息

• TREATMENT OF PROTEINOPATHIES
  申请人：The General Hospital Corporation

  公开号：EP2723384A1

  公开（公告）日：2014-04-30
• MRNA BASED ENZYME REPLACEMENT THERAPY COMBINED WITH A PHARMACOLOGICAL CHAPERONE FOR THE TREATMENT OF LYSOSOMAL STORAGE DISORDERS
  申请人：ModernaTX, Inc.

  公开号：US20220184185A1

  公开（公告）日：2022-06-16

  This disclosure relates to treatment of lysosomal storage disorders, such as Fabry disease or Gaucher disease, with a combination treatment containing (i) an mRNA encoding a lysosomal enzyme deficient in the lysosomal storage disorder, and (ii) a compound that is a glucosylceramide synthase inhibitor or a pharmacological chaperone of the lysosomal enzyme. mRNAs for use in the invention, when administered in vivo, encode the enzyme that is deficient in the lysosomal storage disorder, functional fragments thereof (e.g., those comprising the catalytic domain), or fusion proteins containing the enzyme that is deficient in the lysosomal storage disorder. mRNA therapies can be used to increase and/or restore deficient levels of a lysosomal enzyme's expression and/or activity in subjects.
• US9845327B2
  申请人：——

  公开号：US9845327B2

  公开（公告）日：2017-12-19
• [EN] COMPOSITIONS AND METHODS FOR ENHANCING ENZYME ACTIVITY IN GAUCHER, GM1-GANGLIOSIDOSIS/MORQUIO B DISEASE, AND PARKINSON'S DISEASE<br/>[FR] COMPOSITIONS ET PROCÉDÉS PERMETTANT D'AMÉLIORER L'ACTIVITÉ ENZYMATIQUE DANS LA MALADIE DE GAUCHER, LA GANGLIOSIDOSE À GM1, LA MALADIE DE MORQUIO DE TYPE B ET LA MALADIE DE PARKINSON
  申请人：HOSPITAL FOR SICK CHILDREN

  公开号：WO2009049421A1

  公开（公告）日：2009-04-23

  Therapeutic compositions and methods for treatment of lysosomal storage disorders, such as Gaucher, GM1-gangliosidosis/Morquio B disease and Parkinson's disease are described herein. The compositions comprise compounds having glucocerebrosidase and beta-galactosidase inhibitory activity for use as pharmacological chaperones for mutant forms of the enzyme. Methods of treatment involve providing therapeutically effective amounts of such compounds to subjects in need thereof.
• [EN] COMPOSITIONS AND METHODS FOR ENHANCING ENZYME ACTIVITY IN GAUCHER, GM1 -GANGLIOSIDOSIS/MORQUIO B DISEASE. AND PARKINSON'S DISEASE<br/>[FR] COMPOSITIONS ET PROCÉDÉS PERMETTANT D'AMÉLIORER L'ACTIVITÉ ENZYMATIQUE DANS LA MALADIE DE GAUCHER, LA GANGLIOSIDOSE À GM1, LA MALADIE DE MORQUIO DE TYPE B ET LA MALADIE DE PARKINSON
  申请人：HOSPITAL FOR SICK CHILDREN

  公开号：WO2009049422A1

  公开（公告）日：2009-04-23

  Therapeutic compositions and methods for treatment of lysosomal storage disorders, such as Gaucher, GM1-gangliosidosis/Morquio B disease and Parkinson's disease are described herein. The compositions comprise compounds having glucocerebrosidase and beta-galactosidase inhibitory activity for use as pharmacological chaperones for mutant forms of the enzyme. Methods of treatment involve providing therapeutically effective amounts of 5[(4-methylphenyl) thio] quinazoline-2,4-diamine) to subjects in need thereof.