N-[2-[2-[2-[3-amino-4,5-dihydroxy-6-(hydroxymethyl)oxan-2-yl]oxyethoxy]ethoxy]ethyl]-3-[3-[3-[2-[2-[2-[3-amino-4,5-dihydroxy-6-(hydroxymethyl)oxan-2-yl]oxyethoxy]ethoxy]ethylamino]-3-oxopropoxy]-2-[[3-[2-[2-[2-[3-amino-4,5-dihydroxy-6-(hydroxymethyl)oxan-2-yl]oxyethoxy]ethoxy]ethylamino]-3-oxopropoxy]methyl]-2-methylpropoxy]propanamide

• 分子结构分类: 有机化合物 - 有机氧化合物
• 英文名称: N-[2-[2-[2-[3-amino-4,5-dihydroxy-6-(hydroxymethyl)oxan-2-yl]oxyethoxy]ethoxy]ethyl]-3-[3-[3-[2-[2-[2-[3-amino-4,5-dihydroxy-6-(hydroxymethyl)oxan-2-yl]oxyethoxy]ethoxy]ethylamino]-3-oxopropoxy]-2-[[3-[2-[2-[2-[3-amino-4,5-dihydroxy-6-(hydroxymethyl)oxan-2-yl]oxyethoxy]ethoxy]ethylamino]-3-oxopropoxy]methyl]-2-methylpropoxy]propanamide
• 化学式: C₅₀H₉₆N₆O₂₇
• 分子量: 1213.3
• InChiKey: HUWUEFWUIIUMEC-UHFFFAOYSA-N

计算性质

• 辛醇/水分配系数(LogP)：
  -10.3
• 重原子数：
  83
• 可旋转键数：
  48
• 环数：
  3.0
• sp3杂化的碳原子比例：
  0.94
• 拓扑面积：
  486
• 氢给体数：
  15
• 氢受体数：
  30

文献信息

• COMPLEMENT COMPONENT C5 iRNA COMPOSITIONS AND METHODS OF USE THEREOF
  申请人：Alnylam Pharmaceuticals, Inc.

  公开号：US20150247143A1

  公开（公告）日：2015-09-03

  The invention relates to iRNA, e.g., double-stranded ribonucleic acid (dsRNA), compositions targeting the complement component C5 gene, and methods of using such iRNA, e.g., dsRNA, compositions to inhibit expression of C5 and to treat subjects having a complement component C5-associated disease, e.g., paroxysmal nocturnal hemoglobinuria.

  该发明涉及iRNA，例如双链核糖核酸（dsRNA），针对补体成分C5基因的组合物，以及使用这种iRNA，例如dsRNA，组合物来抑制C5的表达并治疗患有补体成分C5相关疾病的受试者，例如周期性夜间血红蛋白尿。
• COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF MYLIP/IDOL GENE
  申请人：Bettencourt Brian

  公开号：US20130123332A1

  公开（公告）日：2013-05-16

  The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the Mylip/Idol gene, and methods of using such dsRNA compositions to inhibit expression of Mylip/Idol.
• Compositions and Methods for Inhibition of Expression of Apolipoprotein C-III (APOC3) Genes
  申请人：Bettencourt Brian

  公开号：US20140113957A1

  公开（公告）日：2014-04-24

  The invention relates to double-stranded ribonucleic acid (dsRNA) targeting an APOC3 gene, and methods of using the dsRNA to inhibit expression of APOC3.
• ANGIOPOIETIN-LIKE 3 (ANGPTL3) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
  申请人：Alnylam Pharmaceuticals, Inc.

  公开号：US20140179768A1

  公开（公告）日：2014-06-26

  The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the ANGPTL3 gene, as well as methods of inhibiting expression of ANGPTL3 and methods of treating subjects having a disorder of lipid metabolism, such as hyperlipidemia or hypertriglyceridemia, using such dsRNA compositions.
• MODIFIED RNAi AGENTS
  申请人：ALNYLAM PHARMACEUTICALS, INC.

  公开号：US20140288158A1

  公开（公告）日：2014-09-25

  One aspect of the present invention relates to double-stranded RNAi (dsRNA) duplex agent capable of inhibiting the expression of a target gene. The dsRNA duplex comprises one or more motifs of three identical modifications on three consecutive nucleotides in one or both strand, particularly at or near the cleavage site of the strand. Other aspects of the invention relates to pharmaceutical compositions comprising these dsRNA agents suitable for therapeutic use, and methods of inhibiting the expression of a target gene by administering these dsRNA agents, e.g., for the treatment of various disease conditions.