(2R,3R,4S,5S)-2-(6-aminopurin-9-yl)-5-[difluoro(hydroxy)methyl]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,3R,4S,5S)-2-(6-aminopurin-9-yl)-5-[difluoro(hydroxy)methyl]oxolane-3,4-diol
• 化学式: C₁₀H₁₁F₂N₅O₄
• 分子量: 303.22
• InChiKey: IUIHKZFITLQBRP-SOVPELCUSA-N

计算性质

• 辛醇/水分配系数(LogP)：
  -0.3
• 重原子数：
  21
• 可旋转键数：
  2
• 环数：
  3.0
• sp3杂化的碳原子比例：
  0.5
• 拓扑面积：
  140
• 氢给体数：
  4
• 氢受体数：
  10

文献信息

• [EN] POLYNUCLEOTIDES ENCODING JAGGED1 FOR THE TREATMENT OF ALAGILLE SYNDROME<br/>[FR] POLYNUCLÉOTIDES CODANT POUR JAGGED1 POUR LE TRAITEMENT DU SYNDROME D'ALAGILLE
  申请人：MODERNATX INC

  公开号：WO2017201342A1

  公开（公告）日：2017-11-23

  The invention relates to mRNA therapy for the treatment of Alagille syndrome (ALGS), mRNAs for use in the invention, when administered in vivo, encode JAGGED 1 (JAG1), isoforms thereof, functional fragments thereof, and fusion proteins comprising JAG1, mRNAs of the invention are preferably encapsulated in lipid nanoparticles (LNPs) to effect efficient delivery to cells and/or tissues in subjects, when administered thereto. mRNA therapies of the invention increase and/or restore deficient levels of JAG1 expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient JAG1 activity in subjects.

  该发明涉及用于治疗艾拉吉尔综合征（ALGS）的mRNA疗法，该发明中使用的mRNA，在体内给予时，编码JAGGED 1（JAG1）、其同工异构体、其功能性片段以及包含JAG1的融合蛋白，该发明的mRNA最好被封装在脂质纳米颗粒（LNPs）中，以实现对受试者的细胞和/或组织的有效传递，当向受试者施用时。该发明的mRNA疗法增加和/或恢复受试者中JAG1表达和/或活性的不足水平。该发明的mRNA疗法进一步降低与受试者中JAG1活性不足相关的有毒代谢物水平。
• CHIMERIC POLYNUCLEOTIDES
  申请人：Moderna Therapeutics, Inc.

  公开号：EP3041934A1

  公开（公告）日：2016-07-13
• CIRCULAR POLYNUCLEOTIDES
  申请人：Moderna Therapeutics, Inc.

  公开号：EP3169335A2

  公开（公告）日：2017-05-24
• POLYNUCLEOTIDES ENCODING JAGGED1 FOR THE TREATMENT OF ALAGILLE SYNDROME
  申请人：ModernaTX, Inc.

  公开号：EP3458107A1

  公开（公告）日：2019-03-27
• PHARMACEUTICAL COMPOSITIONS FOR TREATING EBOLA VIRUS DISEASE
  申请人：Vecht-Lifshitz Susan Eve

  公开号：US20190282601A1

  公开（公告）日：2019-09-19

  The present invention provides compounds and pharmaceutical compositions adapted to reduce a load of an RNA virus by at least 50%, the virus causing a pathogenic disease in a mammalian subject, the compound adapted to inhibit the formation of S-adenosyl methionine (SAM) in the virus, the compound being a DOT1L inhibitor, wherein the compound has a molecular weight of less than 1000, and a therapeutic index (TI=LD50:ED50) greater than 30 in the mammalian subject.