申请人:Translate Bio MA, Inc.
公开号:EP3533873A1
公开(公告)日:2019-09-04
The disclosure provides multimeric oligonucleotide compounds, comprising two or more target-specific oligonucleotides (e.g., antisense oligonucleotides (ASOs)), each being resistant to cleavage, and linked together by a cleavable linker. In particular, two or more linked target-specific oligonucleotides, each to a different target, allows concomitant inhibition of multiple genes' expression levels, while exhibiting favorable pharmacokinetic and pharmacodynamic properties. Methods of making and uses of the described compounds are also provided
本公开提供了多聚物寡核苷酸化合物,包括两个或多个靶向特异性寡核苷酸(如反义寡核苷酸(ASO)),每个寡核苷酸都具有抗裂解性,并通过可裂解连接体连接在一起。特别是两个或更多链接的靶标特异性寡核苷酸,每个都针对不同的靶标,可以同时抑制多个基因的表达水平,同时表现出良好的药代动力学和药效学特性。还提供了所述化合物的制造方法和用途