15,15-Bis(13,13-dimethyl-5,11-dioxo-2,12-dioxa-6,10-diazatetradecyl)-2,2-dimethyl-4,10,17-trioxo-3,13-dioxa-5,9,16-triazaoctacosan-28-oic acid

• 分子结构分类: 有机化合物 - 脂质和类脂质分子 - 脂肪酰基
• 英文名称: 15,15-Bis(13,13-dimethyl-5,11-dioxo-2,12-dioxa-6,10-diazatetradecyl)-2,2-dimethyl-4,10,17-trioxo-3,13-dioxa-5,9,16-triazaoctacosan-28-oic acid
• 英文别名: 12-[[1,3-bis[3-[3-[(2-methylpropan-2-yl)oxycarbonylamino]propylamino]-3-oxopropoxy]-2-[[3-[3-[(2-methylpropan-2-yl)oxycarbonylamino]propylamino]-3-oxopropoxy]methyl]propan-2-yl]amino]-12-oxododecanoic acid
• 化学式: C₄₉H₉₁N₇O₁₅
• 分子量: 1018.3
• InChiKey: WRPKTGMYIJSECL-UHFFFAOYSA-N

计算性质

• 辛醇/水分配系数(LogP)：
  3.3
• 重原子数：
  71
• 可旋转键数：
  45
• 环数：
  0.0
• sp3杂化的碳原子比例：
  0.84
• 拓扑面积：
  296
• 氢给体数：
  8
• 氢受体数：
  15

文献信息

• [EN] CARBOHYDRATE CONJUGATED RNA AGENTS AND PROCESS FOR THEIR PREPARATION<br/>[FR] AGENTS D'ARN CONJUGUÉS À DES GLUCIDES ET LEUR PROCÉDÉ DE PRÉPARATION
  申请人：ALNYLAM PHARMACEUTICALS INC

  公开号：WO2014025805A1

  公开（公告）日：2014-02-13

  This disclosure relates to an improved process for the preparation of carbohydrate conjugates. The disclosure also relates to carbohydrate conjugated iRNA agents comprising these carbohydrate conjugates, which have improved purity and are advantageous for the in vivo delivery of the iRNA agents.

  这份披露涉及一种改进的碳水化合物结合物制备过程。该披露还涉及包含这些碳水化合物结合物的碳水化合物结合iRNA药剂，其具有改进的纯度，并且对于iRNA药剂的体内输送是有利的。
• CARBOHYDRATE CONJUGATED RNA AGENTS AND PROCESS FOR THEIR PREPARATION
  申请人：ALNYLAM PHARMACEUTICALS, INC.

  公开号：US20150196655A1

  公开（公告）日：2015-07-16

  This disclosure relates to an improved process for the preparation of carbohydrate conjugates. The disclosure also relates to carbohydrate conjugated iRNA agents comprising these carbohydrate conjugates, which have improved purity and are advantageous for the in vivo delivery of the iRNA agents.

  本公开涉及一种改进的糖类结合物制备工艺。本公开还涉及包含这些糖类结合物的糖类结合iRNA药剂，其具有改善的纯度，并且对于体内输送iRNA药剂是有优势的。
• OLIGONUCLEOTIDE COMPOSITIONS AND METHODS OF USE THEREOF
  申请人：VARGEESE Chandra

  公开号：US20200157545A1

  公开（公告）日：2020-05-21

  Among other things, the present disclosure provides designed oligonucleotides, compositions, and methods thereof. In some embodiments, provided oligonucleotide compositions provide improved single-stranded RNA interference and/or RNase H-mediated knockdown. Among other things, the present disclosure encompasses the recognition that structural elements of oligonucleotides, such as base sequence, chemical modifications (e.g., modifications of sugar, base, and/or internucleotidic linkages) or patterns thereof, conjugation with additional chemical moieties, and/or stereochemistry [e.g., stereochemistry of backbone chiral centers (chiral internucleotidic linkages)], and/or patterns thereof, can have significant impact on oligonucleotide properties and activities, e.g., RNA interference (RNAi) activity, stability, delivery, etc. In some embodiments, the present disclosure provides methods for treatment of diseases using provided oligonucleotide compositions, for example, in RNA interference and/or RNase H-mediated knockdown.
• OLIGONUCLEOTIDE COMPOSITIONS AND METHODS THEREOF
  申请人：WAVE LIFE SCIENCES LTD.

  公开号：US20210228615A1

  公开（公告）日：2021-07-29

  Among other things, the present disclosure provides technologies for altering splicing, particularly for increasing inclusion of exons in splicing products. In some embodiments, the present disclosure provides SMN2 oligonucleotides, compositions, and methods thereof. In some embodiments, the present disclosure provides chirally controlled SMN2 oligonucleotide compositions. In some embodiments, provided oligonucleotides and compositions can increase level of an exon 7-containing SMN2 splicing product and/or a gene product thereof. In some embodiments, the present disclosure provides methods for treatment of splicing-related conditions, disorders and diseases. In some embodiments, the present disclosure provides methods for treating SMN2-related conditions, disorders and diseases such as SMA (spinal muscular atrophy) and ALS (amyotrophic lateral sclerosis).