15-(1,2-Dihydroxyethyl)-15-hydroxynonacosane-14,16-dione

• 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: 15-(1,2-Dihydroxyethyl)-15-hydroxynonacosane-14,16-dione
• 化学式: C₃₁H₆₀O₅
• 分子量: 512.8
• InChiKey: WEPMPNRXSPALDE-UHFFFAOYSA-N

计算性质

• 辛醇/水分配系数(LogP)：
  10.3
• 重原子数：
  36
• 可旋转键数：
  28
• 环数：
  0.0
• sp3杂化的碳原子比例：
  0.94
• 拓扑面积：
  94.8
• 氢给体数：
  3
• 氢受体数：
  5

文献信息

• SYNTHESIS AND STRUCTURE OF HIGH POTENCY RNA THERAPEUTICS
  申请人：Arcturus Therapeutics, Inc.

  公开号：US20190002906A1

  公开（公告）日：2019-01-03

  This invention provides expressible polynucleotides, which can express a target protein or polypeptide. Synthetic mRNA constructs for producing a protein or polypeptide can contain one or more 5′ UTRs, where a 5′ UTR may be expressed by a gene of a plant. In some embodiments, a 5′ UTR may be expressed by a gene of a member of Arabidopsis genus. The synthetic mRNA constructs can be used as pharmaceutical agents for expressing a target protein or polypeptide in vivo.

  这项发明提供了可表达的多核苷酸，可以表达目标蛋白质或多肽。用于产生蛋白质或多肽的合成mRNA构建物可以包含一个或多个5' UTR，其中5' UTR可以由植物的基因表达。在某些实施例中，5' UTR可以由拟南芥属植物的基因表达。这些合成mRNA构建物可以用作体内表达目标蛋白质或多肽的药用剂。
• ASIALOGLYCOPROTEIN RECEPTOR MEDIATED DELIVERY OF THERAPEUTICALLY ACTIVE CONJUGATES
  申请人：Arcturus Therapeutics, Inc.

  公开号：US20210060168A1

  公开（公告）日：2021-03-04

  ASGP-R binding molecular conjugates are provided. The conjugates are useful to deliver therapeutically effective amounts of biologically active molecules to target cells and tissues of a subject. Compositions are also provided comprising the molecular conjugates.

  提供ASGP-R结合分子共轭物。这些共轭物可用于将治疗有效量的生物活性分子传递给受试者的靶细胞和组织。还提供包含这些分子共轭物的组合物。
• [EN] PROSTACYCLIN COMPOUNDS, COMPOSITIONS AND METHODS OF USE THEREOF<br/>[FR] COMPOSÉS DE PROSTACYCLINE, COMPOSITIONS ET MÉTHODES D'UTILISATION ASSOCIÉES
  申请人：INSMED INC

  公开号：WO2016176555A1

  公开（公告）日：2016-11-03

  Prostacyclin compounds and compositions comprising the same are provided herein. Specifically, prostacyclin compounds comprising treprostinil covalently linked to a linear C2-C18 alkyl, branched C3-C18 alkyl, linear C2-C18 alkenyl, branched C3-C18 alkenyl, aryl, aryl-C1-C18 alkyl or an amino acid or a peptide (e.g., dipeptide, tripeptide, tetrapeptide) are described, for example, for administration via subcutaneous or intravenous infusion to a patient in need of pulmonary hypertension treatment. The linkage, in one embodiment, is via a carbamate, amide or ester bond. Prostacyclin compounds provided herein can also include at least one hydrogen atom substituted with at least one deuterium atom.

  本文提供了前列环素化合物和包含其的组合物。具体来说，描述了包括曲前列素与线性C2-C18烷基、支链C3-C18烷基、线性C2-C18烯基、支链C3-C18烯基、芳基、芳基-C1-C18烷基或氨基酸或肽（例如二肽、三肽、四肽）共价连接的前列环素化合物，例如，用于经皮下或静脉输注给需要肺动脉高压治疗的患者。在一个实施例中，连接是通过碳酸酯、酰胺或酯键。本文提供的前列环素化合物还可以包括至少一个氢原子被至少一个氘原子取代。
• [EN] ALKENYL SUBSTITUTED 2,5-PIPERAZINEDIONES AND THEIR USE IN COMPOSITIONS FOR DELIVERING AN AGENT TO A SUBJECT OR CELL<br/>[FR] 2,5-PIPÉRAZINEDIONES SUBSTITUÉES PAR UN ALCÉNYLE, ET LEUR UTILISATION DANS DES COMPOSITIONS DESTINÉES À L'ADMINISTRATION D'UN AGENT À UN SUJET OU UNE CELLULE
  申请人：MASSACHUSETTS INST TECHNOLOGY

  公开号：WO2016205691A1

  公开（公告）日：2016-12-22

  Provided herein are compounds of Formula (I), and salts thereof, wherein each instance of RL is independently optionally substituted C6-C40 alkenyl. Further provided are compositions comprising a compound of Formula (I) and an agent. Further provided are methods and kits using the compositions for delivering an agent to a subject or cell and for treating and/or preventing a range of diseases. Further provided are methods of preparing compounds of Formula (I) and precursors thereof.

  本文提供了化合物的结构式（I）及其盐，其中RL的每个实例都是独立的、可选地取代的C6-C40烯基。此外，还提供了包含结构式（I）化合物和药剂的组合物。还提供了使用这些组合物将药剂传递给受试者或细胞，并用于治疗和/或预防一系列疾病的方法和试剂盒。还提供了制备结构式（I）化合物及其前体的方法。
• Biodegradable lipids for delivery of nucleic acids
  申请人：TRANSLATE BIO, INC.

  公开号：US10022455B2

  公开（公告）日：2018-07-17

  The present invention provides, in part, a biodegradable compound of formula I, and sub-formulas thereof: Formula (I) or a pharmaceutically acceptable salt thereof, where each X independently is O or S, each Y independently is O or S, and each R1 independently is defined herein; and a liposome composition comprising the cationic lipid of formula I or a sub-formula thereof, and methods of delivering agents, such as nucleic acids including mRNA, in vivo, by administering to a subject the liposome comprising the cationic lipid of formula I or a sub-formula thereof, where the agent is encapsulated within the liposome.

  本发明提供了一种生物可降解的化合物，其化学式为I，以及其子式：化学式（I）或其药学上可接受的盐，其中每个X独立地为O或S，每个Y独立地为O或S，每个R1独立地在此处定义；以及包含化学式I或其子式的阳离子脂质的脂质体组合物，以及通过向主体给予包含化学式I或其子式的阳离子脂质的脂质体来递送药剂（例如核酸，包括mRNA）的方法，在此过程中，药剂被封装在脂质体内。