4-(benzenesulfonamido)-N-(5-ethyl-1,3,4-thiadiazol-2-yl)benzamide

• 分子结构分类: 有机化合物 - 苯类化合物 - 苯和取代衍生物
• 英文名称: 4-(benzenesulfonamido)-N-(5-ethyl-1,3,4-thiadiazol-2-yl)benzamide
• 化学式: C₁₇H₁₆N₄O₃S₂
• 分子量: 388.5
• InChiKey: YMGXUJCEBOCIIT-UHFFFAOYSA-N

计算性质

• 辛醇/水分配系数(LogP)：
  2.9
• 重原子数：
  26
• 可旋转键数：
  6
• 环数：
  3.0
• sp3杂化的碳原子比例：
  0.12
• 拓扑面积：
  138
• 氢给体数：
  2
• 氢受体数：
  7

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.
• [EN] TREATMENT OF PROTEINOPATHIES<br/>[FR] TRAITEMENT DE PROTÉINOPATHIES
  申请人：GEN HOSPITAL CORP

  公开号：WO2012177997A1

  公开（公告）日：2012-12-27

  The present disclosure provides technologies relating to lysosomal activation. The disclosure provides several strategies for increasing level and/or activity of lysosomal enzyme, and furthermore demonstrates the surprising applicability of such strategies in the treatment and/or prophylaxis of certain proteinopathies. Among other things, the present invention provides methods and compositions for the treatment and/or prophylaxis of proteinopathies other than lysosomal storage diseases through lysosomal activation. In particular, the present disclosure provides methods and compositions for the treatment and/or prophylaxis of neurodegenerative proteinopathies, and in particular those associated with accumulation of α-synuclein. The present disclosure specifically provides methods and compositions for the treatment and/or prophylaxis of Parkinson's disease.