C - Chemistry – Metallurgy – 12 – N
Patent
C - Chemistry, Metallurgy
12
N
C12N 15/11 (2006.01) A61K 31/713 (2006.01) A61K 47/48 (2006.01)
Patent
CA 2685127
The present invention relates to agents, compositions and methods for inhibiting the expression of a target gene, comprising an RNAi agent bearing at least one galactosyl moiety. These are useful for delivering the gene expression inhibiting activity to cells, particularly hepatocytes, and more particularly in therapeutic applications.
La présente invention concerne des agents, compositions et procédés destinés à inhiber l'expression d'un gène cible, comprenant un agent de type ARNi porteur d'au moins un groupe galactosyle. Ils sont utilisables à des fins d'inhibition de l'expression génique au niveau de cellules, en particulier au niveau des hépatocytes, plus particulièrement dans le cadre d'applications thérapeutiques.
Forst Andrea
Hadwiger Philipp
Vornlocher Hans-Peter
Alnylam Pharmaceuticals Inc.
Riches Mckenzie & Herbert Llp
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