[3-[2-Aminoethoxy(hydroxy)phosphoryl]oxy-2-[(5Z,8Z,11Z,14Z)-icosa-5,8,11,14-tetraenoyl]oxypropyl] (5Z,8Z,11Z,14Z)-icosa-5,8,11,14-tetraenoate

• 分子结构分类: 有机化合物 - 脂质和类脂质分子 - 甘油磷脂
• 英文名称: [3-[2-Aminoethoxy(hydroxy)phosphoryl]oxy-2-[(5Z,8Z,11Z,14Z)-icosa-5,8,11,14-tetraenoyl]oxypropyl] (5Z,8Z,11Z,14Z)-icosa-5,8,11,14-tetraenoate
• 化学式: C₄₅H₇₄NO₈P
• 分子量: 788.0
• InChiKey: JTERLNYVBOZRHI-XCHUKFSYSA-N

计算性质

• 辛醇/水分配系数(LogP)：
  8.6
• 重原子数：
  55
• 可旋转键数：
  39
• 环数：
  0.0
• sp3杂化的碳原子比例：
  0.6
• 拓扑面积：
  134
• 氢给体数：
  2
• 氢受体数：
  9

文献信息

• Polynucleotides encoding citrin for the treatment of Citrullinemia type 2
  申请人：ModernaTX, Inc.

  公开号：US10993918B2

  公开（公告）日：2021-05-04

  The invention relates to mRNA therapy for the treatment of Citrullinemia Type 2 (“CTLN2”). mRNAs for use in the invention, when administered in vivo, encode human Citrin, isoforms thereof, functional fragments thereof, and fusion proteins comprising Citrin. mRNAs of the invention are preferably encapsulated in lipid nanoparticles (LNPs) to effect efficient delivery to cells and/or tissues in subjects, when administered thereto. mRNA therapies of the invention increase and/or restore deficient levels of Citrin expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of biomarkers associated with deficient Citrin activity in subjects, namely ammonia and/or triglycerides.

  本发明涉及用于治疗瓜氨酸血症2型（"CTLN2"）的mRNA疗法。用于本发明的mRNA在体内给药时，编码人Citrin、其异构体、其功能片段以及包含Citrin的融合蛋白。本发明的mRNA优选封装在脂质纳米颗粒（LNPs）中，以便在给药时有效地传递到受试者的细胞和/或组织中。本发明的mRNA疗法可增加和/或恢复受试者体内Citrin表达和/或活性的不足水平。本发明的mRNA疗法可进一步降低与受试者体内Citrin活性不足有关的生物标志物水平，即氨和/或甘油三酯。
• COMPOUNDS AND COMPOSITIONS FOR INTRACELLULAR DELIVERY OF AGENTS
  申请人：ModernaTX, Inc.

  公开号：US20210161829A1

  公开（公告）日：2021-06-03

  The disclosure features amino lipids and compositions involving the same. Nanoparticle compositions include an amino lipid as well as additional lipids such as phospholipids, structural lipids, PEG lipids, or a combination thereof. Nanoparticle compositions further including therapeutic and/or prophylactic agents such as RNA are useful in the delivery of therapeutic and/or prophylactic agents to mammalian cells or organs to, for example, regulate polypeptide, protein, or gene expression.