(2R,5R)-2-[2-amino-6-(methylamino)purin-9-yl]-5-(hydroxymethyl)oxolane-3,4-diol

• 分子结构分类: 有机化合物 - 核苷、核苷酸和类似物 - 嘌呤核苷
• 英文名称: (2R,5R)-2-[2-amino-6-(methylamino)purin-9-yl]-5-(hydroxymethyl)oxolane-3,4-diol
• 化学式: C₁₁H₁₆N₆O₄
• 分子量: 296.28
• InChiKey: XTJPMALIPQWXDY-DGPXGRDGSA-N

计算性质

• 辛醇/水分配系数(LogP)：
  -0.7
• 重原子数：
  21
• 可旋转键数：
  3
• 环数：
  3.0
• sp3杂化的碳原子比例：
  0.55
• 拓扑面积：
  152
• 氢给体数：
  5
• 氢受体数：
  9

文献信息

• MODIFIED POLYNUCLEOTIDES FOR THE PRODUCTION OF BIOLOGICS AND PROTEINS ASSOCIATED WITH HUMAN DISEASE
  申请人：Moderna Therapeutics, Inc.

  公开号：EP3520821A1

  公开（公告）日：2019-08-07

  The invention relates to compositions and methods for the preparation, manufacture and therapeutic use of polynucleotides, primary transcripts and mmRNA molecules.

  本发明涉及制备、生产和治疗使用多核苷酸、初级转录本和 mmRNA 分子的组合物和方法。
• Formulation and delivery of modified nucleoside, nucleotide, and nucleic acid compositions
  申请人：ModernaTX, Inc.

  公开号：US10258698B2

  公开（公告）日：2019-04-16

  The present disclosure provides, inter alia, formulation compositions comprising modified nucleic acid molecules which may encode a protein, a protein precursor, or a partially or fully processed form of the protein or a protein precursor. The formulation composition may further include a modified nucleic acid molecule and a delivery agent. The present invention further provides nucleic acids useful for encoding polypeptides capable of modulating a cell's function and/or activity.

  本公开特别提供了包含修饰核酸分子的制剂组合物，修饰核酸分子可编码蛋白质、蛋白质前体或蛋白质或蛋白质前体的部分或完全加工形式。制剂组合物可进一步包括修饰的核酸分子和递送剂。本发明进一步提供了用于编码能够调节细胞功能和/或活性的多肽的核酸。
• Modified polynucleotides for the production of cytoplasmic and cytoskeletal proteins
  申请人：ModernaTX, Inc.

  公开号：US10463751B2

  公开（公告）日：2019-11-05

  The invention relates to compositions including polynucleotides encoding polypeptides which have been chemically modified by replacing the uridines with 1-methyl-pseudouridine to improve one or more of the stability and/or clearance in tissues, receptor uptake and/or kinetics, cellular access by the compositions, engagement with translational machinery, mRNA half-life, translation efficiency, immune evasion, protein production capacity, secretion efficiency, accessibility to circulation, protein half-life and/or modulation of a cell's status, function, and/or activity.

  本发明涉及包括编码多肽的多核苷酸的组合物，这些组合物通过用 1-甲基假尿苷取代尿苷进行化学修饰，以改善组织中的一种或多种稳定性和/或清除率、受体摄取和/或动力学、组合物的细胞通路、与翻译机制的啮合、mRNA 半衰期、翻译效率、免疫逃避、蛋白质生产能力、分泌效率、循环通路、蛋白质半衰期和/或细胞状态、功能和/或活性的调节。
• Modified polynucleotides for the production of secreted proteins
  申请人：ModernaTX, Inc.

  公开号：US10577403B2

  公开（公告）日：2020-03-03

  The invention relates to compositions and methods for the preparation, manufacture and therapeutic use of polynucleotides, primary transcripts and mmRNA molecules.

  本发明涉及制备、生产和治疗使用多核苷酸、初级转录本和 mmRNA 分子的组合物和方法。
• Modified Polynucleotides for the production of biologics and proteins associated with human disease
  申请人：ModernaTX, Inc.

  公开号：US10772975B2

  公开（公告）日：2020-09-15

  The invention relates to a pharmaceutical composition including a plurality of lipid nanoparticles having a mean particle size of between 80 nm and 160 nm and containing an mRNA encoding a polypeptide. The lipid nanoparticles include a cationic lipid, a neutral lipid, a cholesterol, and a PEG lipid. The mRNA contains a 5′-cap, 5′-UTR, a 3′-UTR, and a poly-A region with at least 100 nucleotides.

  本发明涉及一种药物组合物，包括多个脂质纳米颗粒，其平均粒径在 80 纳米到 160 纳米之间，并含有编码多肽的 mRNA。脂质纳米颗粒包括阳离子脂质、中性脂质、胆固醇和 PEG 脂质。mRNA 含有至少 100 个核苷酸的 5′-cap、5′-UTR、3′-UTR 和 poly-A 区域。