ROZEMA, DAVID B.,罗兹马 大卫Ｂ,羅茲馬 大衛Ｂ,WAKEFIELD, DARREN H.,维克菲尔德 戴伦Ｈ,維克菲爾德 戴倫Ｈ,BLOKHIN, ANDREI V.,布洛克希 安卓Ｖ,BENSON, JONATHAN D.,班森 强纳森Ｄ,班森 強納森Ｄ,LI, ZHEN,李,PEI, TAO,裴 涛,裴 濤,FLEITZ, FRED,弗立兹 佛瑞德,弗立,罗兹马 大卫B,羅茲馬 大衛B,维克菲尔德 戴伦H,維克菲爾德 戴倫H,布洛克希 安卓V,班森 强纳森D,班森 強納森D,裴 涛,裴 濤,弗立兹 佛瑞德,弗立茲 佛瑞
申请号：
TW106107454
公开号：
TW201735953A
申请日:
2017.03.07
申请国别(地区):
TW
年份:
2017
代理人:
摘要：
Described are novel targeting ligands that may be linked to compounds, such as therapeutic compounds, that are useful in directing the compounds to the targetin vivo. The targeting ligands disclosed herein can serve to target expression-inhibiting oligomeric compounds, such as RNAi agents, to liver cells to modulate gene expression. The targeting ligands disclosed herein, when conjugated to an expression-inhibiting oligomeric compound, may be used in a variety of applications, as defined in the specification.Compositions including the targeting ligands disclosed herein when linked to expression-inhibiting oligomeric compounds are capable of mediating expression of target nucleic acid sequences in liver cells, such as hepatocytes, which may be useful in the treatment of diseases or conditions that respond to inhibition of gene expression or activity in a cell, tissue, or organism.本文闡述可連接至化合物(例如治療性化合物)之新穎標靶性配體，其可用於將該等化合物引導至活體內標靶。本文所揭示之該等標靶性配體可用於使表現抑制性寡聚化合物(例如RNAi藥劑)靶向肝臟細胞以調節基因表現。本文所揭示之該等標靶性配體在偶聯至表現抑制性寡聚化合物時可用於各種如說明書所定義之應用中。包括本文所揭示之該等標靶性配體之組合物在連接至表現抑制性寡聚化合物時能調介標靶核酸序列於肝臟細胞(例如肝細胞)中之表現，該等組合物可用於治療對細胞、組織或生物體中之基因表現或活性之抑制有反應之疾病或病況。
