5-fluoro-3-(imidazo[1,2-a]pyrimidin-2-yl)-7-(4-methyl-1,4-diazepan-1-yl)-2H-chromen-2-one | 1446330-74-0

• 分子结构分类: 有机化合物 - 脂质和类脂质分子 - 异戊烯醇脂质
• 英文名称: 5-fluoro-3-(imidazo[1,2-a]pyrimidin-2-yl)-7-(4-methyl-1,4-diazepan-1-yl)-2H-chromen-2-one
• 英文别名: 5-Fluoro-3-imidazo[1,2-a]pyrimidin-2-yl-7-(4-methyl-1,4-diazepan-1-yl)chromen-2-one
• CAS: 1446330-74-0
• 化学式: C₂₁H₂₀FN₅O₂
• 分子量: 393.421
• InChiKey: PLCFKSZNYSDGCP-UHFFFAOYSA-N

计算性质

• 辛醇/水分配系数(LogP)：
  3.2
• 重原子数：
  29
• 可旋转键数：
  2
• 环数：
  5.0
• sp3杂化的碳原子比例：
  0.29
• 拓扑面积：
  63
• 氢给体数：
  0
• 氢受体数：
  7

文献信息

• COMPOUNDS FOR TREATING SPINAL MUSCULAR ATROPHY
  申请人：PTC Therapeutics Inc.

  公开号：US20150119380A1

  公开（公告）日：2015-04-30

  Provided herein are compounds, compositions thereof and uses therewith for treating spinal muscular atrophy. In a specific embodiment, provided herein are compounds of a form that may be used to modulate the inclusion of exon 7 of SMN2 into mRNA that is transcribed from the SMN2 gene. In another specific embodiment, provided herein are compounds of a form that may be used to modulate the inclusion of exon 7 of SMN1 into mRNA that is transcribed from the SMN1 gene. In yet another embodiment, provided herein are compounds of a form that may be used to modulate the inclusion of exon 7 of SMN1 and SMN2 into mRNA that is transcribed from the SMN1 and SMN2 genes, respectively.

  本文提供了用于治疗脊髓性肌萎缩症的化合物、其组合物及与其一起使用的用途。在一个具体实施例中，本文提供了一种可以用来调节SMN2基因转录的mRNA中包含外显子7的形式化合物。在另一个具体实施例中，本文提供了一种可以用来调节SMN1基因转录的mRNA中包含外显子7的形式化合物。在另一个实施例中，本文提供了一种可以用来调节SMN1和SMN2基因转录的mRNA中包含外显子7的形式化合物。
• US9617268B2
  申请人：——

  公开号：US9617268B2

  公开（公告）日：2017-04-11
• USRE47689E1
  申请人：——

  公开号：USRE47689E1

  公开（公告）日：2019-11-05
• [EN] COMPOUNDS FOR TREATING SPINAL MUSCULAR ATROPHY<br/>[FR] COMPOSÉS DE TRAITEMENT D'UNE AMYOTROPHIE SPINALE
  申请人：PTC THERAPEUTICS INC

  公开号：WO2013101974A1

  公开（公告）日：2013-07-04

  Provided herein are compounds, compositions thereof and uses therewith for treating spinal muscular atrophy. In a specific embodiment, provided herein are compounds of a form that may be used to modulate the inclusion of exon 7 of SMN2 into mRNA that is transcribed from the SMN2 gene. In another specific embodiment, provided herein are compounds of a form that may be used to modulate the inclusion of exon 7 of SMN1 into mRNA that is transcribed from the SMN1 gene. In yet another embodiment, provided herein are compounds of a form that may be used to modulate the inclusion of exon 7 of SMN1 and SMN2 into mRNA that is transcribed from the SMN1 and SMN2 genes, respectively.
• [EN] METHODS FOR MODULATING THE AMOUNT RNA TRANSCRIPTS<br/>[FR] PROCÉDÉS POUR LA MODULATION DE LA QUANTITÉ DE PRODUITS DE LA TRANSCRIPTION D'ARN
  申请人：PTC THERAPEUTICS INC

  公开号：WO2015095449A1

  公开（公告）日：2015-06-25

  Described herein are methods for modulating the amount of a gene product and compounds for use in such methods. More particularly, described herein are methods for modulating the amount of an RNA transcript or protein product as the result of gene expression and compounds for use in such methods.