[EN] LIPID CONJUGATES FOR THE DELIVERY OF THERAPEUTIC AGENTS<br/>[FR] CONJUGUÉS LIPIDIQUES POUR L'ADMINISTRATION D'AGENTS THÉRAPEUTIQUES
申请人:ARROWHEAD PHARMACEUTICALS INC
公开号:WO2022056273A1
公开(公告)日:2022-03-17
Disclosed herein are compounds according to Formula (I) comprising PK/PD modulators for delivery of oligonucleotide-based agents, e.g., double stranded RNAi agents, to certain cell types, such for example skeletal muscle cells, in vivo. The PK/PD modulators disclosed herein, when conjugated to an oligonucleotide-based therapeutic or diagnostic agent, such as an RNAi agent, can enhance the delivery of the composition to the specified cells being targeted to facilitate the inhibition of gene expression in those cells.
本文公开了公式(I)中的化合物,包括PK/PD调节剂,用于将基于寡核苷酸的制剂(例如双链RNAi制剂)递送到体内某些细胞类型,例如骨骼肌细胞。本文所披露的PK/PD调节剂与基于寡核苷酸的治疗或诊断制剂(如RNAi制剂)结合后,可以增强将该组合物递送到特定的目标细胞,以促进在这些细胞中抑制基因表达。