Methyl 3-[2-[3-[2-[bis(3-methoxy-3-oxopropyl)amino]ethyl-methylamino]propyl-methylamino]ethyl-(3-methoxy-3-oxopropyl)amino]propanoate

• 分子结构分类: 有机化合物 - 有机酸及其衍生物 - 羧酸及其衍生物
• 英文名称: Methyl 3-[2-[3-[2-[bis(3-methoxy-3-oxopropyl)amino]ethyl-methylamino]propyl-methylamino]ethyl-(3-methoxy-3-oxopropyl)amino]propanoate
• 英文别名: methyl 3-[2-[3-[2-[bis(3-methoxy-3-oxopropyl)amino]ethyl-methylamino]propyl-methylamino]ethyl-(3-methoxy-3-oxopropyl)amino]propanoate
• 化学式: C₂₅H₄₈N₄O₈
• 分子量: 532.7
• InChiKey: IMLUQIBNWLGDCF-UHFFFAOYSA-N

计算性质

• 辛醇/水分配系数(LogP)：
  0
• 重原子数：
  37
• 可旋转键数：
  26
• 环数：
  0.0
• sp3杂化的碳原子比例：
  0.84
• 拓扑面积：
  118
• 氢给体数：
  0
• 氢受体数：
  12

文献信息

• Alkenyl substituted 2,5-piperazinediones, compositions, and uses thereof
  申请人：Massachusetts Institute of Technology

  公开号：US10201618B2

  公开（公告）日：2019-02-12

  Provided herein are compounds of Formula (I), and salts thereof, wherein each instance of RL is independently optionally substituted C6-C40 alkenyl. Further provided are compositions comprising a compound of Formula (I) and an agent. Further provided are methods and kits using the compositions for delivering an agent to a subject or cell and for treating and/or preventing a range of diseases. Further provided are methods of preparing compounds of Formula (I) and precursors thereof.

  本文提供了式 (I) 化合物及其盐类，其中 RL 的每个实例均独立地为任选取代的 C6-C40 烯基。还提供了包含式（I）化合物和制剂的组合物。进一步提供的是使用这些组合物的方法和试剂盒，用于向受试者或细胞递送药剂以及治疗和/或预防一系列疾病。进一步提供了制备式（I）化合物及其前体的方法。
• Messenger RNA therapy for the treatment of Friedreich's ataxia
  申请人：Translate Bio, Inc.

  公开号：US10780183B2

  公开（公告）日：2020-09-22

  The present invention provides methods and compositions of treating Friedreich's ataxia (FRDA) based on administering an mRNA encoding a frataxin protein.

  本发明提供了基于施用编码 frataxin 蛋白的 mRNA 来治疗弗里德雷希氏共济失调（FRDA）的方法和组合物。
• Composition and methods for treatment of ornithine transcarbamylase deficiency
  申请人：Translate Bio, Inc.

  公开号：US11167043B2

  公开（公告）日：2021-11-09

  The present invention provides, among other things, methods of treating ornithine transcarbamylase deficiency, including administering to a subject in need of treatment a composition comprising an mRNA encoding an ornithine transcarbamylase protein at a low dose and at an administration interval such that at least one symptom or feature of the OTC deficiency is reduced.

  除其他外，本发明提供了治疗鸟氨酸转氨甲酰酶缺乏症的方法，包括以低剂量和给药间隔向需要治疗的受试者给药包含编码鸟氨酸转氨甲酰酶蛋白的mRNA的组合物，从而减少OTC缺乏症的至少一种症状或特征。
• Treatment of cystic fibrosis by delivery of codon-optimized mRNA encoding CFTR
  申请人：Translate Bio, Inc.

  公开号：US11173190B2

  公开（公告）日：2021-11-16

  The present invention provides, among other things, methods of treating cystic fibrosis, comprising a step of administering to a subject in need of treatment a composition comprising an mRNA encoding a Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein, wherein the mRNA encoding the CFTR protein comprises a polynucleotide sequence at least 80% identical to SEQ ID NO: 1, wherein the mRNA is at a concentration of at least 0.4 mg/mL, and wherein the step of administering comprises inhalation.

  本发明除其他外提供了治疗囊性纤维化的方法，包括向需要治疗的受试者施用包含编码囊性纤维化跨膜传导调节器（CFTR）蛋白的mRNA的组合物的步骤，其中编码CFTR蛋白的mRNA包含与SEQ ID NO: 1至少80%相同的多核苷酸序列，其中mRNA的浓度至少为0.4 mg/mL，施用步骤包括吸入。
• Process of preparing mRNA-loaded lipid nanoparticles
  申请人：Translate Bio, Inc.

  公开号：US11357726B2

  公开（公告）日：2022-06-14

  The present invention provides an improved process for lipid nanoparticle formulation and mRNA encapsulation. In some embodiments, the present invention provides a process of encapsulating messenger RNA (mRNA) in lipid nanoparticles comprising a step of mixing a solution of pre-formed lipid nanoparticles and mRNA at a low concentration.

  本发明提供了一种改进的脂质纳米颗粒配方和mRNA封装方法。在某些实施例中，本发明提供了一种将信使核糖核酸（mRNA）封装在脂质纳米颗粒中的方法，包括在低浓度下混合预形成的脂质纳米颗粒溶液和mRNA的步骤。