2,3-bis(palmitoyloxy)propyl (3-(trimethylammonio)propyl) phosphate | 75658-25-2

• 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: 2,3-bis(palmitoyloxy)propyl (3-(trimethylammonio)propyl) phosphate
• 英文别名: 2,3-Di(hexadecanoyloxy)propyl 3-(trimethylazaniumyl)propyl phosphate
• CAS: 75658-25-2
• 化学式: C₄₁H₈₂NO₈P
• 分子量: 748.078
• InChiKey: BMZAAOMTPSUUFL-UHFFFAOYSA-N

计算性质

• 辛醇/水分配系数(LogP)：
  13.8
• 重原子数：
  51
• 可旋转键数：
  41
• 环数：
  0.0
• sp3杂化的碳原子比例：
  0.95
• 拓扑面积：
  111
• 氢给体数：
  0
• 氢受体数：
  8

反应信息

• 作为产物：
  描述：

  3-(((2,3-bis(palmitoyloxy)propoxy)(2-cyanoethoxy)phosphoryl)oxy)-N,N,N-trimethylpropan-1-aminium chloride 在 N,N-二异丙基乙胺 作用下， 以 乙腈 为溶剂， 反应 16.0h， 以78%的产率得到2,3-bis(palmitoyloxy)propyl (3-(trimethylammonio)propyl) phosphate
  参考文献：
  名称：

  [EN] COMPOSITIONS AND METHODS FOR DELIVERY OF THERAPEUTIC AGENTS
  [FR] COMPOSITIONS ET PROCÉDÉS PERMETTANT D'ADMINISTRER DES AGENTS THÉRAPEUTIQUES

  摘要：

  这项披露提供了改进的基于脂质的组合物，包括脂质纳米粒子组合物，以及它们用于体内传递药剂的方法，包括核酸和蛋白质。这些组合物不受加速血清清除的影响，并且它们在体内具有改进的毒性特性。

  公开号：

  WO2017099823A1

文献信息

• Nguyen Hoang Phuong et al., Comptes Rendus des Seances de l'Academie des Sciences, Serie C: Sciences Chimiques, 1976, vol. 283, p. 323 - 325
  作者：Nguyen Hoang Phuong et al.

  DOI：——

  日期：——
• COMPOSITIONS AND METHODS FOR DELIVERY OF AGENTS
  申请人：ModernaTX, Inc.

  公开号：US20180028664A1

  公开（公告）日：2018-02-01

  This disclosure provides improved lipid-based compositions, including lipid nanoparticle compositions, and methods of use thereof for delivering agents in vivo including nucleic acids and proteins. These compositions are not subject to accelerated blood clearance and they have an improved toxicity profile in vivo.
• [EN] POLYNUCLEOTIDES ENCODING α-GALACTOSIDASE A FOR THE TREATMENT OF FABRY DISEASE<br/>[FR] POLYNUCLÉOTIDES CODANT POUR L'Α-GALACTOSIDASE A POUR LE TRAITEMENT DE LA MALADIE DE FABRY
  申请人：MODERNATX INC

  公开号：WO2017201328A1

  公开（公告）日：2017-11-23

  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.
• [EN] POLYNUCLEOTIDES ENCODING PORPHOBILINOGEN DEAMINASE FOR THE TREATMENT OF ACUTE INTERMITTENT PORPHYRIA<br/>[FR] POLYNUCLÉOTIDES CODANT POUR LA PORPHOBILINOGÈNE DÉSAMINASE DESTINÉS AU TRAITEMENT DE LA PORPHYRIE INTERMITTENTE AIGUË
  申请人：MODERNATX INC

  公开号：WO2017201346A1

  公开（公告）日：2017-11-23

  The invention relates to mRNA therapy for the treatment of Acute Intermittent Porphyria (AIP). mRNAs for use in the invention, when administered in vivo, encode human porphobilinogen deaminase (PBGD), isoforms thereof, functional fragments thereof, and fusion proteins comprising PBGD. mRNAs of the invention are preferably encapsulated in lipid nanoparticles (LNPs) to affect efficient delivery to cells and/or tissues in subjects, when administered thereto. mRNA therapies of the invention increase and/or restore deficient levels of PBGD expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient PBGD activity in subjects, namely porphobilinogen and aminolevulinate (PBG and ALA).
• [EN] POLYRIBONUCLEOTIDES CONTAINING REDUCED URACIL CONTENT AND USES THEREOF<br/>[FR] POLYRIBONUCLÉOTIDES CONTENANT UNE TENEUR RÉDUITE EN URACILE ET UTILISATIONS ASSOCIÉES
  申请人：MODERNATX INC

  公开号：WO2017201317A1

  公开（公告）日：2017-11-23

  The invention related to polyribonucleotides comprising an open reading frame of linked nucleosides encoding a polypeptide of interest (e.g., a therapeutic polypeptide), isoforms thereof, functional fragments thereof, and fusion proteins comprising the polypeptide. In some embodiments, the open reading frame is sequence-optimized. In particular embodiments, the invention provides sequence-optimized polyribonucleotides comprising nucleotides encoding the sequence of the polypeptide of interest, or sequence having high sequence identity with those sequence optimized polyribonucleotides.