Votoplam | 2407849-89-0

• 分子结构分类: 有机化合物 - 有机杂环化合物 - 唑类
• 英文名称: Votoplam
• 英文别名: 2-[3-(2,2,6,6-tetramethylpiperidin-4-yl)triazolo[4,5-c]pyridazin-6-yl]-5-(triazol-2-yl)phenol
• CAS: 2407849-89-0
• 化学式: C₂₁H₂₅N₉O
• 分子量: 419.5
• InChiKey: ICZNVPJUHBURBG-UHFFFAOYSA-N

计算性质

• 辛醇/水分配系数(LogP)：
  1.9
• 重原子数：
  31
• 可旋转键数：
  3
• 环数：
  5.0
• sp3杂化的碳原子比例：
  0.43
• 拓扑面积：
  120
• 氢给体数：
  2
• 氢受体数：
  8

反应信息

• 作为产物：
  描述：

  6-chloro-N3-(2,2,6,6-tetramethylpiperidin-4-yl)pyridazine-3,4-diamine 在 盐酸 、 tris-(dibenzylideneacetone)dipalladium(0) 、 四丁基氟化铵 、 三乙胺 、 sodium nitrite 作用下， 生成 Votoplam
  参考文献：
  名称：

  HETEROCYCLIC AND HETEROARYL COMPOUNDS FOR TREATING HUNTINGTON'S DISEASE

  摘要：

  Disclosed are quinoline and quinazoline compounds which modulate the activity of the gated ion channels. Compounds that modulate these gated ion channels are useful in the treatment of diseases and disorders related to pain, inflammation, the neurological system, the gastrointestinal system and genitourinary system. Preferred compounds include quinoline or quinazoline derivatives substituted at the 4-position via N(H), C(O) or O moieties.

  公开号：

  US20240132509A1

文献信息

• NOVEL RNA TRANSCRIPT
  申请人：PTC Therapeutics, Inc.

  公开号：US20220162610A1

  公开（公告）日：2022-05-26

  An alternatively spliced intronic sequence is disclosed, the splicing of which can be induced in the presence of a small molecule, as described herein.
• [EN] NOVEL RNA TRANSCRIPT<br/>[FR] NOUVEAU TRANSCRIT D'ARN
  申请人：[en]PTC THERAPEUTICS INC.

  公开号：WO2022103980A1

  公开（公告）日：2022-05-19

  As described herein, an alternatively spliced intronic sequence is induced in the presence of a small molecule, e.g., Compound (I). Thus, in the presence of Compound (I), an intronic sequence is converted into an "intron-derived exon" that can be spliced into the mature mRNA transcript, leading to a frameshift in the mRNA open reading frame and in frame premature stop codons. The premature termination of translation triggers nonsense mediated mRNA decay and a concomitant reduction in the amount of protein encoded by the mRNA. Conversely, in the absence of Compound (I), the intronic sequence is spliced out of the pre-mRNA without causing a change to the mRNA's reading frame. In one aspect, Compound (I) can be 2-[3-(2,2,6,6-tetramethylpiperidin-4-yl)-3H- [1,2,3]triazolo[4,5-c]pyridazin-6-yl]-5-(2H-1,2,3-triazol-2-yl)phenol having the structure of: HTT-C3 Compound (I) can be orally administered with broad biodistribution for the treatment of Huntington's Disease by production of a small molecule-induced alternatively spliced transcript.