6-[2-[4-[Bis(4-fluorophenyl)methyl]piperidin-1-yl]ethyl]-7-methyl-[1,3]thiazolo[3,2-a]pyrimidin-5-one

• 分子结构分类: 有机化合物 - 苯类化合物 - 苯和取代衍生物
• 英文名称: 6-[2-[4-[Bis(4-fluorophenyl)methyl]piperidin-1-yl]ethyl]-7-methyl-[1,3]thiazolo[3,2-a]pyrimidin-5-one
• 化学式: C₂₇H₂₇F₂N₃OS
• 分子量: 479.6
• InChiKey: FHVQKMXOVBAZDC-UHFFFAOYSA-N

计算性质

• 辛醇/水分配系数(LogP)：
  5.2
• 重原子数：
  34
• 可旋转键数：
  6
• 环数：
  5.0
• sp3杂化的碳原子比例：
  0.33
• 拓扑面积：
  61.2
• 氢给体数：
  0
• 氢受体数：
  6

文献信息

• METHOD FOR ENHANCING FOLDING AND TRANSPORT OF MISFOLDED GLUCOCEREBROSIDASE
  申请人：The Hospital for Sick Children

  公开号：US20140249233A1

  公开（公告）日：2014-09-04

  Therapeutic compositions and methods for treatment of late-onset Gaucher disease are described herein. The compositions comprise compounds having activity as pharmacological chaperones for mutant forms of the beta-glucocerebrosidase. Methods of treatment involve providing therapeutically effective amounts of such compositions to subjects in need thereof.
• COMPOSITIONS AND USES THEREOF
  申请人：University of Virginia Patent Foundation

  公开号：US20200231551A1

  公开（公告）日：2020-07-23

  The disclosure is directed to compounds of the formula (IA), (I)-(V) and others disclosed herein and uses of such compounds.
• METHODS AND COMPOSITIONS FOR TREATING PAX6-DEFICIENCY RELATED DISEASE
  申请人：INSERM (INSTITUT NATIONAL DE LA SANTÉ ET DE AL RECHERCHE MÉDICALE)

  公开号：US20220265659A1

  公开（公告）日：2022-08-25

  The present invention relates to an agent for increasing PAX6 gene and/or protein expression in a subject in need thereof. The insertion of a nonsense mutation found in patients allowed inventors to identify altered gene expression of PAX6 target genes, enhanced cell adhesion and reduced cell migration and proliferation. Remarkably, these parameters were rescued by recombinant PAX6 protein. Using this unique cellular model in which they inserted multiple copies of PAX6 responsive elements and tdTomato, they screened a FDA-approved library and identified in a first hit, 3 small compounds that enhance production of PAX6 protein while two of them did not modulate PAX6 gene expression. More particularly, they observed that the small compounds efficiently rescue in vitro migration of mutated limbal cells. Moreover, in the art, it is known that a decrease of PAX6 leads to an induction of diabetes. Small compounds identified by the inventors are suitable to treat PAX6-related deficiencies diseases such as aniridia and diabetes.