1-hexoxy-N,N-dimethyl-3-[(9Z,12Z)-octadeca-9,12-dienoxy]propan-2-amine

• 分子结构分类: 有机化合物 - 有机氮化合物
• 英文名称: 1-hexoxy-N,N-dimethyl-3-[(9Z,12Z)-octadeca-9,12-dienoxy]propan-2-amine
• 化学式: C₂₉H₅₇NO₂
• 分子量: 451.8
• InChiKey: QNHQHPALHHOYJN-QGLGPCELSA-N

计算性质

• 辛醇/水分配系数(LogP)：
  9.6
• 重原子数：
  32
• 可旋转键数：
  25
• 环数：
  0.0
• sp3杂化的碳原子比例：
  0.86
• 拓扑面积：
  21.7
• 氢给体数：
  0
• 氢受体数：
  3

文献信息

• [EN] NUCLEIC ACID CONJUGATES AND USES THEREOF<br/>[FR] CONJUGUÉS DE TYPE ACIDE NUCLÉIQUE ET LEURS UTILISATIONS
  申请人：TRANSLATE BIO MA INC

  公开号：WO2018013525A1

  公开（公告）日：2018-01-18

  Provided herein are conjugates comprising targeting moieties such as sugars, folates and cell-penetrating peptides, which can be used for the improved delivery of agents (e.g., nucleic acids, such as oligonucleotides or mRNAs, or other agents) to cells. The invention provides conjugates and compounds comprising targeting moieties, methods for preparing the same, and intermediates useful in their preparation. In another aspect, the present invention provides formulations (e.g., pharmaceutical compositions) comprising the targetting moiety-containing conjugates and compounds. The present invention also provides methods for delivering agents (e.g., nucleic acids such as oligonucleotides or mRNAs) to a cell, methods for treating and/or preventing a disease or condition in a subject, and methods for modulating gene expression in a cell or a subject. Further, provided herein are kits comprising the conjugates, or formulations thereof; and kits for the preparation of conjugates described herein.

  本文提供了包含靶向基团的共轭物，如糖类、叶酸和穿膜肽，可用于改善药物（例如核酸，如寡核苷酸或mRNA，或其他药物）对细胞的传递。该发明提供了包含靶向基团的共轭物和化合物，以及其制备方法和制备过程中有用的中间体。另一方面，本发明提供了包含靶向基团的共轭物和化合物的配方（例如，药物组合物）。本发明还提供了将药物（例如核酸，如寡核苷酸或mRNA）传递至细胞的方法，用于治疗和/或预防受试者疾病或状况的方法，以及用于调节细胞或受试者基因表达的方法。此外，本文提供了包含这些共轭物或其配方的试剂盒；以及用于制备本文所述共轭物的试剂盒。
• [EN] COMPOSITIONS AND METHODS FOR DELIVERY OF THERAPEUTIC AGENTS<br/>[FR] COMPOSITIONS ET PROCÉDÉS PERMETTANT D'ADMINISTRER DES AGENTS THÉRAPEUTIQUES
  申请人：MODERNATX INC

  公开号：WO2017099823A1

  公开（公告）日：2017-06-15

  This disclosure provides improved lipid-based compositions, including lipid nanoparticle compositions, and methods of use thereof for delivering agents in vivo including nucleic acids and proteins. These compositions are not subject to accelerated blood clearance and they have an improved toxicity profile in vivo.

  这项披露提供了改进的基于脂质的组合物，包括脂质纳米粒子组合物，以及它们用于体内传递药剂的方法，包括核酸和蛋白质。这些组合物不受加速血清清除的影响，并且它们在体内具有改进的毒性特性。
• Enhanced immunogenicity of mRNA with co-encoded adjuvant sequences
  申请人：PRESIDENT AND FELLOWS OF HARVARD COLLEGE

  公开号：US10736957B2

  公开（公告）日：2020-08-11

  Described herein are in vitro-transcribed (IVT) RNA molecules comprising, a 5′ cap structure, a coding region encoding an antigen polypeptide, an immunostimulatory RNA sequence, and a poly(A) tail.

  本文描述的体外转录（IVT）RNA 分子包括：5′帽结构、编码抗原多肽的编码区、免疫刺激 RNA 序列和聚（A）尾。
• Polynucleotides encoding interleukin-12 (IL12) and uses thereof
  申请人：ModernaTX, Inc.

  公开号：US11000573B2

  公开（公告）日：2021-05-11

  The present disclosure relates to polynucleotides comprising an open reading frame of linked nucleosides encoding human interleukin-12 (IL12), functional fragments thereof, and fusion proteins comprising IL12. In some embodiments, the open reading frame is sequence-optimized. In particular embodiments, the disclosure provides sequence-optimized polynucleotides comprising nucleotides encoding the polypeptide sequence of human IL12, or sequences having high sequence identity with those sequence optimized polynucleotides.

  本公开涉及包含编码人白细胞介素-12（IL12）的连接核苷开放阅读框的多核苷酸、其功能片段以及包含IL12的融合蛋白。在某些实施方案中，开放阅读框是序列优化的。在特定的实施方案中，本公开提供了序列优化的多核苷酸，包括编码人IL12多肽序列的核苷酸，或与这些序列优化的多核苷酸具有高序列同一性的序列。
• Nucleic acid conjugates and uses thereof
  申请人：Translate Bio MA, Inc.

  公开号：US11253601B2

  公开（公告）日：2022-02-22

  Provided herein are conjugates comprising targeting moieties such as sugars, folates and cell-penetrating peptides, which can be used for the improved delivery of agents (e.g., nucleic acids, such as oligonucleotides or mRNAs, or other agents) to cells. The invention provides conjugates and compounds comprising targeting moieties, methods for preparing the same, and intermediates useful in their preparation. In another aspect, the present invention provides formulations (e.g., pharmaceutical compositions) comprising the targeting moiety-containing conjugates and compounds. The present invention also provides methods for delivering agents (e.g., nucleic acids such as oligonucleotides or mRNAs) to a cell, methods for treating and/or preventing a disease or condition in a subject, and methods for modulating gene expression in a cell or a subject. Further, provided herein are kits comprising the conjugates, or formulations thereof; and kits for the preparation of conjugates described herein.

  本发明提供了包含靶向分子（如糖、叶酸盐和细胞穿透肽）的共轭物，可用于改善向细胞递送制剂（如核酸，如寡核苷酸或 mRNA，或其他制剂）。本发明提供了包含靶向分子的共轭物和化合物、制备方法以及用于制备它们的中间体。在另一方面，本发明提供了包含靶向分子的共轭物和化合物的制剂（如药物组合物）。本发明还提供了向细胞递送制剂（如核酸，如寡核苷酸或 mRNA）的方法，治疗和/或预防受试者疾病或病症的方法，以及调节细胞或受试者基因表达的方法。此外，本文还提供了包含共轭物或其制剂的试剂盒；以及制备本文所述共轭物的试剂盒。