12-[[1,3-Bis[3-[3-[5-[3-acetamido-4,5-dihydroxy-6-(hydroxymethyl)oxan-2-yl]oxypentanoylamino]propylamino]-3-oxopropoxy]-2-[[3-[3-[5-[3-acetamido-4,5-dihydroxy-6-(hydroxymethyl)oxan-2-yl]oxypentanoylamino]propylamino]-3-oxopropoxy]methyl]propan-2-yl]amino]-12-oxododecanoic acid

• 分子结构分类: 有机化合物 - 脂质和类脂质分子 - 糖脂
• 英文名称: 12-[[1,3-Bis[3-[3-[5-[3-acetamido-4,5-dihydroxy-6-(hydroxymethyl)oxan-2-yl]oxypentanoylamino]propylamino]-3-oxopropoxy]-2-[[3-[3-[5-[3-acetamido-4,5-dihydroxy-6-(hydroxymethyl)oxan-2-yl]oxypentanoylamino]propylamino]-3-oxopropoxy]methyl]propan-2-yl]amino]-12-oxododecanoic acid
• 英文别名: 12-[[1,3-bis[3-[3-[5-[3-acetamido-4,5-dihydroxy-6-(hydroxymethyl)oxan-2-yl]oxypentanoylamino]propylamino]-3-oxopropoxy]-2-[[3-[3-[5-[3-acetamido-4,5-dihydroxy-6-(hydroxymethyl)oxan-2-yl]oxypentanoylamino]propylamino]-3-oxopropoxy]methyl]propan-2-yl]amino]-12-oxododecanoic acid
• 化学式: C₇₃H₁₃₀N₁₀O₃₀
• 分子量: 1627.9
• InChiKey: PIVNBUXKJFLQQG-UHFFFAOYSA-N

计算性质

• 辛醇/水分配系数(LogP)：
  -5.8
• 重原子数：
  113
• 可旋转键数：
  63
• 环数：
  3.0
• sp3杂化的碳原子比例：
  0.85
• 拓扑面积：
  593
• 氢给体数：
  20
• 氢受体数：
  30

文献信息

• [EN] CARBOHYDRATE CONJUGATED RNA AGENTS AND PROCESS FOR THEIR PREPARATION<br/>[FR] AGENTS D'ARN CONJUGUÉS À DES GLUCIDES ET LEUR PROCÉDÉ DE PRÉPARATION
  申请人：ALNYLAM PHARMACEUTICALS INC

  公开号：WO2014025805A1

  公开（公告）日：2014-02-13

  This disclosure relates to an improved process for the preparation of carbohydrate conjugates. The disclosure also relates to carbohydrate conjugated iRNA agents comprising these carbohydrate conjugates, which have improved purity and are advantageous for the in vivo delivery of the iRNA agents.

  这份披露涉及一种改进的碳水化合物结合物制备过程。该披露还涉及包含这些碳水化合物结合物的碳水化合物结合iRNA药剂，其具有改进的纯度，并且对于iRNA药剂的体内输送是有利的。
• Oligonucleotide compositions and methods thereof
  申请人：WAVE LIFE SCIENCES LTD.

  公开号：US10450568B2

  公开（公告）日：2019-10-22

  Among other things, the present disclosure relates to designed oligonucleotides, compositions, and methods thereof. In some embodiments, provided oligonucleotide compositions provide altered splicing of a transcript. In some embodiments, provided oligonucleotide compositions have low toxicity. In some embodiments, provided oligonucleotide compositions provide improved protein binding profiles. In some embodiments, provided oligonucleotide compositions have improved delivery. In some embodiments, provided oligonucleotide compositions have improved uptake. In some embodiments, the present disclosure provides methods for treatment of diseases using provided oligonucleotide compositions.

  除其他外，本公开涉及设计的寡核苷酸、组合物及其方法。在一些实施方案中，所提供的寡核苷酸组合物能改变转录本的剪接。在一些实施方案中，所提供的寡核苷酸组合物具有低毒性。在一些实施方案中，所提供的寡核苷酸组合物提供了改进的蛋白质结合概况。在一些实施方案中，所提供的寡核苷酸组合物具有更好的传递性。在一些实施方案中，所提供的寡核苷酸组合物改善了吸收。在一些实施方案中，本公开提供了使用所提供的寡核苷酸组合物治疗疾病的方法。
• Oligonucleotides, compositions and methods thereof
  申请人：WAVE LIFE SCIENCES LTD.

  公开号：US11013757B2

  公开（公告）日：2021-05-25

  The present disclosure pertains to the recognition that immune responses mediated by CpG oligonucleotides can be affected by the stereochemistry of modified internucleotidic linkages such as phosphorothioates. In some embodiments, the present disclosure relates to chirally controlled CpG oligonucleotide compositions comprising CpG oligonucleotides comprising multiple modified internucleotidic linkages such as phosphorothioate linkages, wherein the oligonucleotides comprise one or more CpG region motifs having defined stereochemistry patterns of chiral internucleotidic linkages. In some embodiments, CpG oligonucleotides comprising one or more CpG region motifs are capable of agonizing an immune response. In some embodiments, CpG oligonucleotides comprising one or more CpG region motifs are antagonistic. Methods for making and using chirally controlled CpG oligonucleotide compositions are also described. In some embodiments, no immune modulation is desired, and the present disclosure provides methods of identifying chirally controlled oligonucleotide compositions which have decreased immune modulation.

  本公开涉及这样一种认识，即 CpG 寡核苷酸介导的免疫反应可受硫代磷酸酯等修饰的核苷酸间连接的立体化学的影响。在一些实施方案中，本公开涉及手性控制的 CpG 寡核苷酸组合物，该组合物包含CpG 寡核苷酸，该CpG 寡核苷酸包含多个修饰的核苷酸间连接，如硫代磷酸酯连接，其中寡核苷酸包含一个或多个 CpG 区域基序，该 CpG 区域基序具有手性核苷酸间连接的确定立体化学模式。在一些实施方案中，包含一个或多个 CpG 区域基团的 CpG 寡核苷酸能够激动免疫反应。在某些实施方案中，包含一个或多个 CpG 区域基团的 CpG 寡核苷酸具有拮抗作用。还描述了制作和使用啁啾控制 CpG 寡核苷酸组合物的方法。在某些实施方案中，不需要免疫调节，本公开提供了确定免疫调节性降低的手性控制寡核苷酸组合物的方法。
• OLIGONUCLEOTIDE COMPOSITIONS AND METHODS THEREOF
  申请人：WAVE LIFE SCIENCES LTD.

  公开号：US20190127733A1

  公开（公告）日：2019-05-02

  Among other things, the present disclosure relates to designed oligonucleotides, compositions, and methods thereof. In some embodiments, provided oligonucleotide compositions provide altered splicing of a transcript. In some embodiments, provided oligonucleotide compositions have low toxicity. In some embodiments, provided oligonucleotide compositions provide improved protein binding profiles. In some embodiments, provided oligonucleotide compositions have improved delivery. In some embodiments, provided oligonucleotide compositions have improved uptake. In some embodiments, the present disclosure provides methods for treatment of diseases using provided oligonucleotide compositions.
• OLIGONUCLEOTIDES, COMPOSITIONS AND METHODS THEREOF
  申请人：WAVE LIFE SCIENCES LTD.

  公开号：US20190209604A1

  公开（公告）日：2019-07-11

  The present disclosure pertains to the recognition that immune responses mediated by CpG oligonucleotides can be affected by the stereochemistry of modified internucleotidic linkages such as phosphorothioates. In some embodiments, the present disclosure relates to chirally controlled CpG oligonucleotide compositions comprising CpG oligonucleotides comprising multiple modified internucleotidic linkages such as phosphorothioate linkages, wherein the oligonucleotides comprise one or more CpG region motifs having defined stereochemistry patterns of chiral internucleotidic linkages. In some embodiments, CpG oligonucleotides comprising one or more CpG region motifs are capable of agonizing an immune response. In some embodiments, CpG oligonucleotides comprising one or more CpG region motifs are antagonistic. Methods for making and using chirally controlled CpG oligonucleotide compositions are also described. In some embodiments, no immune modulation is desired, and the present disclosure provides methods of identifying chirally controlled oligonucleotide compositions which have decreased immune modulation.