LNA OLIGONUCLEOTIDE CARBOHYDRATE CONJUGATES Russian patent published in 2018 - IPC C12N15/113 A61K31/712 

FIELD: biochemistry.

SUBSTANCE: invention relates to the field of biochemistry. Described is a group of inventions which includes a conjugate that reduces the level of liver expressed RNA, comprising an antisense LNA oligomer and conjugate moiety covalently linked thereto, comprising a group directed at the asialoglycoprotein receptor and a pharmaceutical composition, containing said conjugate in an effective amount. In one embodiment of the invention, the antisense LNA oligomer is 10-22 nucleotide gapmer, and the group directed at the asialoglycoprotein receptor is a galactose cluster with two or three N-acetylgalactosamines. Liver expressed RNA is an mRNA selected from the group consisting of: factor VII, C6 of the complement component, Bcl2, transthyretin (TTR), PCSK9, apolipoprotein B (ApoB), glucagon receptor (GCGR), C-reactive protein (CRP), diacylglycerol acyltransferase type 2 (DGAT2), glucocorticoid receptor (GCCR), phosphatase and tensin homolog (PTEN), tyrosine phosphatase PTP1B, sodium-glucose cotransporter type 2 (SGLT2) and superoxide dismutase SOD1 or RNA virus, for example, hepatitis C virus or hepatitis B.

EFFECT: invention widens the range agents for inhibiting RNA expression.

21 cl, 21 dwg, 12 ex, 17 tbl