Hexadecanoic acid, 3,7,11,15-tetramethyl-,(1r)-1-[[[(2-aminoethoxy)hydroxyphosphinyl]oxy]methyl]-1,2-ethanediylester

• 分子结构分类: 有机化合物 - 脂质和类脂质分子 - 异戊烯醇脂质
• 英文名称: Hexadecanoic acid, 3,7,11,15-tetramethyl-,(1r)-1-[[[(2-aminoethoxy)hydroxyphosphinyl]oxy]methyl]-1,2-ethanediylester
• 英文别名: 2-azaniumylethyl 2,3-bis(3,7,11,15-tetramethylhexadecanoyloxy)propyl phosphate
• 化学式: C₄₅H₉₀NO₈P
• 分子量: 804.2
• InChiKey: XLPHMKQBBCKEFO-UHFFFAOYSA-N

计算性质

• 辛醇/水分配系数(LogP)：
  12.6
• 重原子数：
  55
• 可旋转键数：
  39
• 环数：
  0.0
• sp3杂化的碳原子比例：
  0.96
• 拓扑面积：
  134
• 氢给体数：
  2
• 氢受体数：
  9

文献信息

• [EN] siRNAs WITH AT LEAST TWO LIGANDS AT DIFFERENT ENDS<br/>[FR] ARNSI POSSÉDANT AU MOINS DEUX LIGANDS À DEUX EXTRÉMITÉS DISTINCTES
  申请人：SILENCE THERAPEUTICS GMBH

  公开号：WO2019193189A1

  公开（公告）日：2019-10-10

  There is provided inter alia a conjugate for inhibiting expression of a target gene in a cell, said conjugate comprising a nucleic acid portion and ligand portions, said nucleic acid portion comprising at least one duplex region that comprises at least a portion of a first RNA strand and at least a portion of a second RNA strand that is at least partially complementary to the first strand, wherein said first strand is at least partially complementary to at least a portion of RNA transcribed from said target gene, said ligand portions comprising a linker moiety and a targeting ligand for in vivo targeting of cells and being conjugated exclusively to the 3' and/or 5' ends of one or both RNA strands, wherein the 5' end of the first RNA strand is not conjugated, wherein: (i) the second RNA strand is conjugated at the 5' end to the targeting ligand, and wherein (a) the second RNA strand is also conjugated at the 3' end to the targeting ligand and the 3' end of the first RNA strand is not conjugated; or (b) the first RNA strand is conjugated at the 3' end to the targeting ligand and the 3' end of the second RNA strand is not conjugated; or (c) both the second RNA strand and the first RNA strand are also conjugated at the 3' ends to the targeting ligand; or (ii) both the second RNA strand and the first RNA strand are conjugated at the 3' ends to the targeting ligand and the 5' end of the second RNA strand is not conjugated.

  提供了一种用于抑制细胞中靶基因表达的共轭物，该共轭物包括核酸部分和配体部分，所述核酸部分包括至少一个双链区域，该区域包括至少部分第一RNA链和至少部分第二RNA链，该第二RNA链至少部分与第一链互补，其中所述第一链至少部分与从所述靶基因转录的RNA的至少部分互补，所述配体部分包括连接子基团和用于体内细胞靶向的靶向配体，并且仅连接到一个或两个RNA链的3'和/或5'末端，其中第一RNA链的5'末端未连接，其中：(i) 第二RNA链在5'末端连接到靶向配体，其中(a) 第二RNA链还在3'末端连接到靶向配体，且第一RNA链的3'末端未连接；或(b) 第一RNA链在3'末端连接到靶向配体，且第二RNA链的3'末端未连接；或(c) 第二RNA链和第一RNA链的3'末端均连接到靶向配体；或(ii) 第二RNA链和第一RNA链的3'末端均连接到靶向配体，且第二RNA链的5'末端未连接。
• [EN] NUCLEIC ACIDS FOR INHIBITING EXPRESSION OF PROS1 IN A CELL<br/>[FR] ACIDES NUCLÉIQUES POUR INHIBER L'EXPRESSION DE PROS1 DANS UNE CELLULE
  申请人：UNIV BERN

  公开号：WO2020225301A1

  公开（公告）日：2020-11-12

  The invention relates to nucleic acid products that interfere with PROS1 gene expression or inhibit its expression. The nucleic acids are particularly for use in the treatment, prevention or reduction of risk of suffering from a bleeding disorder.

  该发明涉及干扰PROS1基因表达或抑制其表达的核酸产品。这些核酸特别用于治疗、预防或减少患出血障碍的风险。
• LIPID NANOPARTICLE FORMULATION
  申请人：Silence Therapeutics (London) Ltd

  公开号：EP3315125A1

  公开（公告）日：2018-05-02

  The present invention relates to formulations comprising cationic lipids, including novel cationic lipids, in combination with other lipid components such as cholesterol, fusogenic co-lipids, and PEG-lipids to form lipid nanoparticles with nucleotides to facilitate cellular uptake.

  本发明涉及由阳离子脂质（包括新型阳离子脂质）与其他脂质成分（如胆固醇、融合性助脂质和 PEG 脂质）组合而成的制剂，以形成带有核苷酸的脂质纳米颗粒，从而促进细胞吸收。
• PRODUCTS AND COMPOSITIONS FOR INHIBITING EXPRESSION OF A TARGET GENE
  申请人：Silence Therapeutics GmbH

  公开号：EP3550021A1

  公开（公告）日：2019-10-09

  The present invention relates to products and compositions and their uses. In particular the invention relates to nucleic acid products that interfere with gene expression or inhibits its expression and therapeutic uses such as for the treatment of disease and disorders.

  本发明涉及产品和组合物及其用途。特别是本发明涉及干扰基因表达或抑制基因表达的核酸产品以及治疗用途，如治疗疾病和失调。
• PRODUCTS AND COMPOSITIONS
  申请人：Silence Therapeutics GmbH

  公开号：EP3550022A1

  公开（公告）日：2019-10-09

  The present invention relates to products and compositions and their uses. In particular the invention relates to nucleic acid products that interfere with target gene expression or inhibit target gene expression and therapeutic uses of such products.

  本发明涉及产品和组合物及其用途。本发明尤其涉及干扰靶基因表达或抑制靶基因表达的核酸产品及其治疗用途。