((2S,6R)-6-(5-methyl-2,4-dioxo-3,4-dihydropyrimidin-1(2H)-yl)-4-tritylmorpholin-2-yl)methyl (4-(pyrrolidin-1-yl)piperidin-1-yl)phosphonochloridate hydrochloride | 1407898-61-6

• 分子结构分类: 有机化合物 - 苯类化合物 - 三苯基化合物
• 英文名称: ((2S,6R)-6-(5-methyl-2,4-dioxo-3,4-dihydropyrimidin-1(2H)-yl)-4-tritylmorpholin-2-yl)methyl (4-(pyrrolidin-1-yl)piperidin-1-yl)phosphonochloridate hydrochloride
• 英文别名: 1-[(2R,6S)-6-[[chloro-(4-pyrrolidin-1-ylpiperidin-1-yl)phosphoryl]oxymethyl]-4-tritylmorpholin-2-yl]-5-methylpyrimidine-2,4-dione;hydrochloride
• CAS: 1407898-61-6
• 化学式: C₃₈H₄₅ClN₅O₅P*ClH
• 分子量: 754.693
• InChiKey: YESAYZYKQOJCIS-HFWOIIOLSA-N

计算性质

• 辛醇/水分配系数(LogP)：
  6.38
• 重原子数：
  51
• 可旋转键数：
  10
• 环数：
  7.0
• sp3杂化的碳原子比例：
  0.42
• 拓扑面积：
  94.7
• 氢给体数：
  2
• 氢受体数：
  8

反应信息

• 作为产物：
  描述：

  4-吡咯烷-1-基哌啶 在 N-甲基咪唑 、 2,6-二甲基吡啶 、 三氯氧磷 作用下， 以 二氯甲烷 为溶剂， 反应 7.0h， 生成 ((2S,6R)-6-(5-methyl-2,4-dioxo-3,4-dihydropyrimidin-1(2H)-yl)-4-tritylmorpholin-2-yl)methyl (4-(pyrrolidin-1-yl)piperidin-1-yl)phosphonochloridate hydrochloride
  参考文献：
  名称：

  [EN] OLIGONUCLEOTIDE ANALOGUES HAVING MODIFIED INTERSUBUNIT LINKAGES AND/OR TERMINAL GROUPS
  [FR] ANALOGUES OLIGONUCLÉOTIDIQUES AYANT DES LIAISONS MODIFIÉES ENTRE SOUS-UNITÉS ET/OU DES GROUPES TERMINAUX MODIFIÉS

  摘要：

  公开号：

  WO2011150408A3

文献信息

• FUNCTIONALLY-MODIFIED OLIGONUCLEOTIDES AND SUBUNITS THEREOF
  申请人：Sarepta Therapeutics, Inc.

  公开号：US20140330006A1

  公开（公告）日：2014-11-06

  Functionally-modified oligonucleotide analogues comprising modified intersubunit linkages and/or modified 3′ and/or 5′-end groups are provided. The disclosed compounds are useful for the treatment of diseases where inhibition of protein expression or correction of aberrant mRNA splice products produces beneficial therapeutic effects.

  提供了包含修改的亚单位间连接和/或修改的3'和/或5'-末端基团的功能修饰寡核苷酸类似物。所公开的化合物对于治疗需要抑制蛋白质表达或纠正异常mRNA剪接产物以产生有益治疗效果的疾病是有用的。
• OLIGONUCLEOTIDE ANALOGUES HAVING MODIFIED INTERSUBUNIT LINKAGES AND/OR TERMINAL GROUPS
  申请人：Hanson Gunnar J.

  公开号：US20120065169A1

  公开（公告）日：2012-03-15

  Oligonucleotide analogues comprising modified intersubunit linkages and/or modified 3′ and/or 5′-end groups are provided. The disclosed compounds are useful for the treatment of diseases where inhibition of protein expression or correction of aberrant mRNA splice products produces beneficial therapeutic effects.

  提供了包含修改的亚单位连接和/或修改的3'和/或5'-末端基团的寡核苷酸类似物。所披露的化合物对于治疗需要抑制蛋白质表达或纠正异常mRNA剪接产物以产生有益治疗效果的疾病非常有用。
• PEPTIDE OLIGONUCLEOTIDE CONJUGATES
  申请人：Hanson Gunnar J.

  公开号：US20120289457A1

  公开（公告）日：2012-11-15

  Oligonucleotide analogues conjugated to carrier peptides are provided. The disclosed compounds are useful for the treatment of various diseases, for example diseases where inhibition of protein expression or correction of aberrant mRNA splice products produces beneficial therapeutic effects.

  提供了与载体肽共轭的寡核苷酸类似物。所公开的化合物可用于治疗各种疾病，例如抑制蛋白质表达或校正异常的mRNA剪接产物可产生有益的治疗效果的疾病。
• COMPOSITIONS AND METHODS FOR TREATING DUCHENNE MUSCULAR DYSTROPHY AND RELATED DISORDERS
  申请人：Sarepta Therapeutics, Inc.

  公开号：EP3858993A1

  公开（公告）日：2021-08-04

  The present disclosure relates to compositions and methods for the treatment of Duchenne muscular dystrophy and related disorders. Modified antisense oligomers are disclosed for the treatment of Duchenne muscular dystrophy and related disorders.

  本公开涉及治疗杜氏肌营养不良症及相关疾病的组合物和方法。本发明公开了用于治疗杜氏肌营养不良症及相关疾病的修饰反义寡聚物。
• Functionally-modified oligonucleotides and subunits thereof
  申请人：Sarepta Therapeutics, Inc.

  公开号：US10344281B2

  公开（公告）日：2019-07-09

  Functionally-modified oligonucleotide analogues comprising modified intersubunit linkages and/or modified 3′ and/or 5′-end groups are provided. The disclosed compounds are useful for the treatment of diseases where inhibition of protein expression or correction of aberrant mRNA splice products produces beneficial therapeutic effects.

  本研究提供了功能修饰的寡核苷酸类似物，这些类似物包含修饰的亚基间连接和/或修饰的 3′和/或 5′末端基团。所公开的化合物可用于治疗抑制蛋白质表达或校正异常 mRNA 剪接产物产生有益治疗效果的疾病。