N-[(5S)-6-oxo-5-[5-(1,2,3,4,6-pentahydroxyhexoxy)pentanoylamino]heptyl]-5-(1,2,3,4,6-pentahydroxyhexoxy)pentanamide

• 分子结构分类: 有机化合物 - 脂质和类脂质分子 - 脂肪酰基
• 英文名称: N-[(5S)-6-oxo-5-[5-(1,2,3,4,6-pentahydroxyhexoxy)pentanoylamino]heptyl]-5-(1,2,3,4,6-pentahydroxyhexoxy)pentanamide
• 化学式: C₂₉H₅₆N₂O₁₅
• 分子量: 672.8
• InChiKey: SLFAOWWGTKAOFG-PIFBZSMXSA-N

计算性质

• 辛醇/水分配系数(LogP)：
  -4.7
• 重原子数：
  46
• 可旋转键数：
  29
• 环数：
  0.0
• sp3杂化的碳原子比例：
  0.9
• 拓扑面积：
  296
• 氢给体数：
  12
• 氢受体数：
  15

文献信息

• Transthyretin (TTR) iRNA compositions and methods of use thereof for treating or preventing TTR-associated diseases
  申请人：Alnylam Pharmaceuticals, Inc.

  公开号：US10208307B2

  公开（公告）日：2019-02-19

  The present invention provides iRNA agents, e.g., double stranded iRNA agents, that target the transthyretin (TTR) gene and methods of using such iRNA agents for treating or preventing TTR-associated diseases.

  本发明提供了靶向转甲状腺素（TTR）基因的 iRNA 制剂，如双链 iRNA 制剂，以及使用这种 iRNA 制剂治疗或预防 TTR 相关疾病的方法。
• Compositions and methods for inhibiting expression of the ALAS1 gene
  申请人：ALNYLAM PHARMACEUTICALS, INC.

  公开号：US10400239B2

  公开（公告）日：2019-09-03

  The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the ALAS1 gene, and methods of using such dsRNA compositions to alter (e.g., inhibit) expression of ALAS1.

  本发明涉及靶向 ALAS1 基因的双链核糖核酸（dsRNA）组合物，以及使用这种 dsRNA 组合物改变（如抑制）ALAS1 表达的方法。
• Methods and compositions for treating a proprotein convertase subtilisin kexin (PCSK9) gene-associated disorder
  申请人：Alnylam Pharmaceuticals, Inc.

  公开号：US10851377B2

  公开（公告）日：2020-12-01

  The invention relates to methods of inhibiting the expression of a PCSK9 gene in a subject, as well as therapeutic and prophylactic methods for treating subjects having a lipid disorder, such as a hyperlipidemia using RNAi agents, e.g., double-stranded RNAi agents, targeting the PCSK9 gene.

  本发明涉及抑制受试者体内 PCSK9 基因表达的方法，以及使用 RNAi 制剂（如双链 RNAi 制剂）靶向 PCSK9 基因治疗受试者血脂紊乱（如高脂血症）的治疗和预防方法。
• Polynucleotide agents targeting factor XII (hageman factor) (F12) and methods of use thereof
  申请人：Alnylam Pharmaceuticals, Inc.

  公开号：US10883107B2

  公开（公告）日：2021-01-05

  The invention relates to polynucleotide agents targeting Factor XII (F12) gene, and methods of using such polynucleotide agents to inhibit expression of Factor XII and to treat subjects having a Factor XII-associated disease, e.g., heredity angioedema (HAE), prekallikrein deficiency, malignant essential hypertension, hypertension, end stage renal disease, or Fletcher Factor Deficiency.

  本发明涉及靶向因子 XII（F12）基因的多核苷酸制剂，以及使用此类多核苷酸制剂抑制因子 XII 的表达和治疗患有因子 XII 相关疾病（如遗传性血管性水肿（HAE）、前胰激肽原缺乏症、恶性本质性高血压、高血压、终末期肾病或弗莱彻因子缺乏症）的受试者的方法。
• Carbohydrate conjugates as delivery agents for oligonucleotides
  申请人：ALNYLAM PHARMACEUTICALS, INC.

  公开号：US11110174B2

  公开（公告）日：2021-09-07

  The present invention provides a phosphorothioate-modified oligonucleotide comprising a structure shown below: The present invention also provides a phosphorothioate-modified oligonucleotide comprising a structure having formula (CIII):

  本发明提供了一种硫代磷酸酯修饰的寡核苷酸，其结构如下所示： 本发明还提供了一种硫代磷酸酯修饰的寡核苷酸，其结构为式(CIII)：