N¹-(2-(4-(2-(di((Z)-octadec-9-en-1-yl)amino)ethyl)piperazin-1-yl)ethyl)-N¹,N²,N²-tri((Z)-octadec-9-en-1-yl)ethane-1,2-diamine

• 分子结构分类: 有机化合物 - 有机杂环化合物 - 二嗪烷类
• 英文名称: N¹-(2-(4-(2-(di((Z)-octadec-9-en-1-yl)amino)ethyl)piperazin-1-yl)ethyl)-N¹,N²,N²-tri((Z)-octadec-9-en-1-yl)ethane-1,2-diamine
• 英文别名: N'-[2-[4-[2-[bis[(Z)-octadec-9-enyl]amino]ethyl]piperazin-1-yl]ethyl]-N,N,N'-tris[(Z)-octadec-9-enyl]ethane-1,2-diamine
• 化学式: C₁₀₀H₁₉₅N₅
• 分子量: 1467.68
• InChiKey: OSLZZUOTOFCDCM-GLPREEDZSA-N

计算性质

• 辛醇/水分配系数(LogP)：
  39.7
• 重原子数：
  105
• 可旋转键数：
  89
• 环数：
  1.0
• sp3杂化的碳原子比例：
  0.9
• 拓扑面积：
  16.2
• 氢给体数：
  0
• 氢受体数：
  5

文献信息

• [EN] COMPOUNDS AND COMPOSITIONS FOR INTRACELLULAR DELIVERY OF AGENTS<br/>[FR] COMPOSÉS ET COMPOSITIONS POUR L'ADMINISTRATION INTRACELLULAIRE D'AGENTS THÉRAPEUTIQUES ET/OU PROPHYLACTIQUES
  申请人：MODERNATX INC

  公开号：WO2017112865A1

  公开（公告）日：2017-06-29

  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.

  该披露涉及氨基脂质和相关组合物。纳米粒子组合物包括氨基脂质以及额外的脂质，如磷脂、结构脂质、PEG脂质或二者的组合。纳米粒子组合物还包括治疗和/或预防剂，如RNA，可用于将治疗和/或预防剂传递至哺乳动物细胞或器官，例如，调节多肽、蛋白质或基因表达。
• POLYNUCLEOTIDES ENCODING ALPHA-GALACTOSIDASE A FOR THE TREATMENT OF FABRY DISEASE
  申请人：ModernaTX, Inc.

  公开号：EP3896164A1

  公开（公告）日：2021-10-20

  The invention relates to mRNA therapy for the treatment of Fabry disease. mRNAs for use in the invention, when administered in vivo, encode human the α-galactosidase A (GLA), isoforms thereof, functional fragments thereof, and fusion proteins comprising GLA. 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 GLA expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient GLA activity in subjects, namely Gb3 and lyso-Gb3.

  本发明涉及治疗法布里病的mRNA疗法。用于本发明的mRNA在体内给药时，编码人的α-半乳糖苷酶A（GLA）、其同工型、其功能片段和包含GLA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疗法可提高和/或恢复受试者体内不足的GLA表达和/或活性水平。本发明的mRNA疗法可进一步降低与受试者体内GLA活性不足有关的毒性代谢物，即Gb3和溶菌酶Gb3的水平。
• Compounds and compositions for intracellular delivery of agents
  申请人：ModernaTX, Inc.

  公开号：US10195156B2

  公开（公告）日：2019-02-05

  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.

  本公开的特征是氨基脂质和涉及氨基脂质的组合物。纳米颗粒组合物包括氨基脂质以及其他脂质，如磷脂、结构脂质、PEG 脂质或其组合。纳米颗粒组合物进一步包括治疗和/或预防剂，如 RNA，可用于向哺乳动物细胞或器官递送治疗和/或预防剂，以调节多肽、蛋白质或基因表达等。
• Polynucleotides encoding relaxin
  申请人：ModernaTX, Inc.

  公开号：US10730924B2

  公开（公告）日：2020-08-04

  The invention relates to mRNA therapy for the treatment of fibrosis and/or cardiovascular disease. mRNAs for use in the invention, when administered in vivo, encode human relaxin, isoforms thereof, functional fragments thereof, and fusion proteins comprising relaxin. 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 relaxin expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient relaxin activity in subjects.

  本发明涉及治疗纤维化和/或心血管疾病的mRNA疗法。用于本发明的mRNA在体内给药时，可编码人松弛素、其同工酶、其功能片段以及包含松弛素的融合蛋白。本发明的 mRNA 优选封装在脂质纳米颗粒 (LNP) 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 疗法可提高和/或恢复受试者体内不足的松弛素表达水平和/或活性。
• Polynucleotides encoding galactose-1-phosphate uridylyltransferase for the treatment of galactosemia type 1
  申请人：ModernaTX, Inc.

  公开号：US11001861B2

  公开（公告）日：2021-05-11

  The invention relates to mRNA therapy for the treatment of galactosemia type 1 (Gal-1). mRNAs for use in the invention, when administered in vivo, encode human galactose-1-phosphate uridylyltransferase (GALT), isoforms thereof, functional fragments thereof, and fusion proteins comprising GALT. 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 GALT expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient GALT activity in subjects, namely galactose-1-phosphate (Gal-1-P).

  本发明涉及用于治疗1型半乳糖血症（Gal-1）的mRNA疗法。用于本发明的mRNA在体内给药时，编码人半乳糖-1-磷酸尿苷酰转移酶（GALT）、其异构体、其功能片段以及包含GALT的融合蛋白。本发明的 mRNA 最好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/或恢复受试者体内不足的 GALT 表达和/或活性水平。