4-[4-(2,3-Dihydro-1,4-benzodioxin-6-ylsulfonyl)piperazin-1-yl]-2-thiophen-2-ylquinazoline

• 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-[4-(2,3-Dihydro-1,4-benzodioxin-6-ylsulfonyl)piperazin-1-yl]-2-thiophen-2-ylquinazoline
• 化学式: C₂₄H₂₂N₄O₄S₂
• 分子量: 494.6
• InChiKey: ADVNIXIJJUZLHD-UHFFFAOYSA-N

计算性质

• 辛醇/水分配系数(LogP)：
  3.7
• 重原子数：
  34
• 可旋转键数：
  4
• 环数：
  6.0
• sp3杂化的碳原子比例：
  0.25
• 拓扑面积：
  122
• 氢给体数：
  0
• 氢受体数：
  9

文献信息

• 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.