(2R,3S,4R,5R)-5-(2,6-diaminopurin-9-yl)-2-(hydroxymethyl)-4-methoxyoxolan-3-ol

• 分子结构分类: 有机化合物 - 核苷、核苷酸和类似物 - 嘌呤核苷
• 英文名称: (2R,3S,4R,5R)-5-(2,6-diaminopurin-9-yl)-2-(hydroxymethyl)-4-methoxyoxolan-3-ol
• 化学式: C₁₁H₁₆N₆O₄
• 分子量: 296.28
• InChiKey: JLWUWXCKSOIFPS-GAWUUDPSSA-N

计算性质

• 辛醇/水分配系数(LogP)：
  -1
• 重原子数：
  21
• 可旋转键数：
  3
• 环数：
  3.0
• sp3杂化的碳原子比例：
  0.55
• 拓扑面积：
  155
• 氢给体数：
  4
• 氢受体数：
  9

文献信息

• [EN] RNAI AGENTS FOR INHIBITING EXPRESSION OF BETA-ENAC, COMPOSITIONS THEREOF, AND METHODS OF USE<br/>[FR] AGENTS D'ARNI POUR INHIBER L'EXPRESSION DE BETA-ENAC, LEURS COMPOSITIONS ET PROCÉDÉS D'UTILISATION
  申请人：ARROWHEAD PHARMACEUTICALS INC

  公开号：WO2021086995A1

  公开（公告）日：2021-05-06

  Described are RNAi agents, compositions that include RNAi agents, and methods for inhibition of a beta-ENaC (SCNN1B) gene. The beta-ENaC RNAi agents and RNAi agent conjugates disclosed herein inhibit the expression of a beta-ENaC gene. Pharmaceutical compositions that include one or more beta-ENaC RNAi agents, optionally with one or more additional therapeutics, are also described. Delivery of the described beta-ENaC RNAi agents to epithelial cells, such as pulmonary epithelial cells, in vivo, provides for inhibition of beta-ENaC gene expression and a reduction in ENaC activity, which can provide a therapeutic benefit to subjects, including human subjects, for the treatment of various diseases including chronic obstructive pulmonary disease (COPD).

  描述了RNAi药剂、包含RNAi药剂的组合物，以及抑制β-ENaC（SCNN1B）基因的方法。本文披露的β-ENaC RNAi药剂和RNAi药剂结合物抑制了β-ENaC基因的表达。还描述了包含一个或多个β-ENaC RNAi药剂的药物组合物，可选地与一个或多个额外治疗药物一起使用。将所述的β-ENaC RNAi药剂传递至上皮细胞，如体内的肺上皮细胞，可实现抑制β-ENaC基因表达和减少ENaC活性，这可以为受试者，包括人类受试者，提供治疗益处，用于治疗包括慢性阻塞性肺疾病（COPD）在内的各种疾病。
• RNAi agents and compositions for inhibiting expression of angiopoietin-like 3 (ANGPTL3), and methods of use
  申请人：Arrowhead Pharmaceuticals, Inc.

  公开号：US10995335B2

  公开（公告）日：2021-05-04

  The present disclosure relates to RNAi agents, e.g., double stranded RNAi agents, able to inhibit Angiopoietin-like 3 (also called ANGPTL3, ANGPL3, angiopoietin-like protein 3) gene expression, and compositions that include ANGPTL3 RNAi agents. Also disclosed are methods of use of ANGPTL3 RNAi agents and compositions. The ANGPTL3 RNAi agents disclosed herein may be conjugated to targeting ligands to facilitate the delivery to cells, including to hepatocytes. Pharmaceutical compositions that include one or more ANGPTL3 RNAi agents, optionally with one or more additional therapeutics, are described. Delivery of the ANGPTL3 RNAi agents in vivo provides for inhibition of ANGPTL3 gene expression, and can result in lower triglycerides and/or cholesterol levels in the subject. The RNAi agents can be used in methods of treatment of ANGPTL3-related diseases and disorders, including cardiometabolic diseases such as hypertriglyceridemia and hyperlipidemia.

  本公开涉及能够抑制血管生成素样3（又称ANGPTL3、ANGPL3、血管生成素样蛋白3）基因表达的RNAi制剂，如双链RNAi制剂，以及包括ANGPTL3 RNAi制剂的组合物。还公开了ANGPTL3 RNAi制剂和组合物的使用方法。本文公开的 ANGPTL3 RNAi 制剂可与靶向配体连接，以促进向细胞（包括肝细胞）的递送。本文描述了包括一种或多种 ANGPTL3 RNAi 制剂的药物组合物，可选择与一种或多种其它治疗药物一起使用。在体内递送 ANGPTL3 RNAi 制剂可抑制 ANGPTL3 基因的表达，从而降低受试者体内甘油三酯和/或胆固醇的水平。RNAi 制剂可用于治疗 ANGPTL3 相关疾病和失调，包括高甘油三酯血症和高脂血症等心脏代谢疾病。
• ARTIFICIAL NUCLEIC ACID MOLECULES
  申请人：CureVac AG

  公开号：EP3341482B1

  公开（公告）日：2022-07-06
• RNAI AGENTS AND COMPOSITIONS FOR INHIBITING EXPRESSION OF ANGIOPOIETIN-LIKE 3 (ANGPTL3), AND METHODS OF USE
  申请人：Arrowhead Pharmaceuticals, Inc.

  公开号：EP3681513A1

  公开（公告）日：2020-07-22
• RNAI AGENTS FOR INHIBITING EXPRESSION OF BETA-ENAC, COMPOSITIONS THEREOF, AND METHODS OF USE
  申请人：Arrowhead Pharmaceuticals, Inc.

  公开号：EP4051798A1

  公开（公告）日：2022-09-07