(2R,3R,4R,5S)-1-丁基-2-(羟基甲基)-3,4,5-哌啶三醇盐酸盐(1:1) | 210110-90-0

• 物质功能分类: 生物化工 - 生化试剂 - 激动剂抑制剂
• 分子结构分类: 有机化合物 - 有机杂环化合物 - 哌啶类
• 中文名称: (2R,3R,4R,5S)-1-丁基-2-(羟基甲基)-3,4,5-哌啶三醇盐酸盐(1:1)
• 中文别名: N-丁基脱氧野MY霉素盐酸盐
• 英文名称: miglustat hydrochloride
• 英文别名: N-butyldeoxynojirimycin hydrochloride；N-butyldeoxynojirimycin；NB-DNJ；(2R,3R,4R,5S)-1-butyl-2-(hydroxymethyl)piperidine-3,4,5-triol;hydrochloride
• CAS: 210110-90-0
• 化学式: C₁₀H₂₁NO₄*ClH
• 分子量: 255.742
• InChiKey: QPAFAUYWVZMWPR-ZSOUGHPYSA-N

物化性质

• 熔点：
  169-172°C
• 溶解度：
  可溶于DMSO（高达20mg/ml）或水（高达20mg/ml）

计算性质

• 辛醇/水分配系数(LogP)：
  -4.34
• 重原子数：
  16
• 可旋转键数：
  4
• 环数：
  1.0
• sp3杂化的碳原子比例：
  1.0
• 拓扑面积：
  84.2
• 氢给体数：
  5
• 氢受体数：
  6

安全信息

• 危险品标志：
  Xi
• 危险性防范说明：
  P261,P305+P351+P338
• 危险性描述：
  H302,H315,H319,H335
• 储存条件：
  2-8℃

制备方法与用途

生物活性方面，米格拉酯盐酸盐是一种葡萄糖神经酰胺合成酶抑制剂，用于治疗I型戈谢病。

反应信息

• 作为反应物：
  描述：

  (2R,3R,4R,5S)-1-丁基-2-(羟基甲基)-3,4,5-哌啶三醇盐酸盐(1:1) 在 1,8-二氮杂双环[5.4.0]十一碳-7-烯 作用下， 以99.5%的产率得到米格鲁他
  参考文献：
  名称：

  Process For The Preparation Of High Purity Miglustat

  摘要：

  一种制备和分离结晶米格鲁斯塔的方法，不需要使用柱层析或离子交换纯化。结晶米格鲁斯塔具有高纯度，熔点为128°C，且在133°C处具有一个吸热峰。

  公开号：

  US20160168092A1

文献信息

• USE OF PRODRUGS TO AVOID GI MEDIATED ADVERSE EVENTS
  申请人：Franklin Richard

  公开号：US20120202756A1

  公开（公告）日：2012-08-09

  The present invention relates to prodrugs of a wide variety of drugs and pharmaceutical compositions containing such prodrugs. Methods for minimizing locally mediated (from within the gut lumen) adverse gastrointestinal events associated with the underivatised drug and increasing the sustainment of plasma drug levels with the aforementioned prodrugs are also provided. Thus, the present invention relates to the use of prodrugs of a wide diversity of drugs (other than opioids) to transiently inactivate them and so reduce directly, locally mediated adverse gastrointestinal (GI) side-effects normally evident after administration of the parent compound. Additionally, such prodrugs may confer improved pharmacokinetics.

  本发明涉及各种药物的前药和含有这些前药的制药组合物。还提供了一种方法，用于最小化与未衍生药物相关的局部介导（来自肠腔内）不良胃肠事件，并使用上述前药增加血浆药物水平的持续性。因此，本发明涉及使用各种药物（除阿片类药物外）的前药，以短暂地使它们失活，从而减少通常在给予原始化合物后出现的直接、局部介导的不良胃肠（GI）副作用。此外，这些前药可能提供改善的药代动力学。
• PROCESS FOR THE PREPARATION OF HIGH PURITY MIGLUSTAT
  申请人：Navinta, LLC

  公开号：EP3031800A1

  公开（公告）日：2016-06-15

  The present invention provides a process for the preparation and isolation of crystalline miglustat without the use of a column chromatography or ion exchange purification. The crystalline miglustat has a high purity and a melting point of 128 °C and an endothermic peak is 133 °C.

  本发明提供了一种无需使用柱层析或离子交换纯化法制备和分离晶体米格司他的工艺。晶体米格司他纯度高，熔点为 128 °C，内热峰为 133 °C。
• COMPOUNDS HAVING CASPASE INHIBITORY ACTIVITY, PHARMACEUTICAL AGENT CONTAINING SAID COMPOUNDS AND FOR TREATING OR PREVENTING CORNEAL ENDOTHELIAL SYMPTOMS, DISORDERS, OR DISEASES, AND APPLICATION OF SAID PHARMACEUTICAL AGENT
  申请人：The Doshisha

  公开号：EP3639855A1

  公开（公告）日：2020-04-22

  The present invention provides a composition for treating or preventing corneal endothelial symptoms, disorders, or diseases that are attributed to TGF-β signaling in corneal endothelial cells. Provided by the present invention is a composition that includes a compound and that is for treating or preventing endothelial symptoms, disorders, or diseases, wherein, when the compound comes into contact with immortalized cells of Fuchs' corneal endothelial dystrophy, (i) said immortalized cells exhibit a cell survival rate (%) of approximately 90% or more after being cultured for 24-28 hours in Dulbecco's modified Eagle medium (DMEM) + 2% fetal bovine serum (FBS) + 1% penicillin/streptomycin (P/S), and (ii) the ratio of caspase 3/7 activity (%) in the presence of TGF-β with respect to said cellular survival rate (%) is at most 0.8 after being cultured for 24-28 hours in Dulbecco's modified Eagle medium (DMEM) + 2% fetal bovine serum (FBS) + 1% penicillin/streptomycin (P/S).

  本发明提供了一种用于治疗或预防角膜内皮症状、失调或疾病的组合物，这些症状、失调或疾病归因于角膜内皮细胞中的TGF-β信号传导。本发明提供了一种包含化合物的组合物，该组合物用于治疗或预防内皮症状、紊乱或疾病，其中，当该化合物与福氏角膜内皮营养不良症的永生化细胞接触时、(i) 在杜氏改良鹰培养基（DMEM）+2% 胎牛血清（FBS）+1% 青霉素/链霉素（P/S）中培养 24-28 小时后，所述永生化细胞表现出约 90% 或以上的细胞存活率（%），以及 (ii) 在 TGF-β 存在下，caspase 3/7 活性（%）与所述细胞存活率（%）之比至多为 0.在杜氏改良老鹰培养基（DMEM）+2% 胎牛血清（FBS）+1% 青霉素/链霉素（P/S）中培养 24-28 小时后，细胞存活率（%）最多为 0.8。
• Methods and compositions for treating multiple sclerosis
  申请人：University of Miami

  公开号：US10703811B2

  公开（公告）日：2020-07-07

  The compositions and methods described herein include agents that inhibit inflammasome signaling in the mammal such as antibodies directed against inflammasome components used alone or in combination with extracellular vesicle uptake inhibitor(s). Also described herein are compositions and methods of use thereof for treating multiple sclerosis.

  本文所述的组合物和方法包括抑制哺乳动物体内炎性体信号转导的制剂，如单独使用或与细胞外囊泡摄取抑制剂联合使用的针对炎性体成分的抗体。本文还描述了用于治疗多发性硬化症的组合物及其使用方法。
• Methods for treating lung inflammation with an anti-ASC antibody
  申请人：University of Miami

  公开号：US10961306B2

  公开（公告）日：2021-03-30

  The compositions and methods described herein include agents that inhibit inflammasome signaling in the mammal such as antibodies directed against inflammasome components used alone or in combination with extracellular vesicle uptake inhibitor(s). Also described herein are compositions and methods of use thereof for treating multiple sclerosis.

  本文所述的组合物和方法包括抑制哺乳动物体内炎性体信号转导的制剂，如单独使用或与细胞外囊泡摄取抑制剂联合使用的针对炎性体成分的抗体。本文还描述了用于治疗多发性硬化症的组合物及其使用方法。