I-RNA COMPOSITIONS FOR SERPIN AND METHODS OF USE THEREOF Russian patent published in 2019 - IPC C07H21/04 C12N15/113 

FIELD: biotechnologies.

SUBSTANCE: invention relates to the biotechnology. Described is an agent based on double-stranded ribonucleic acid (dsRNA) for inhibiting serpin c1 expression, wherein said dsRNA agent comprises a sense chain and an antisense chain, wherein said antisense strand comprises a complementarity region which contains at least 19 serially arranged nucleotides, differing by not more than 3 nucleotides from nucleotide sequence 5’-UUGAAGUAAAUGGUGUUAACCAG-3’(SEQ ID NO: 562), wherein said sense chain independently has a length of at least 19 nucleotides and where a ligand comprising one or more derivatives of N-acetyl galactosamine (GalNAc), conjugated with 3'-end of said sense chain of said dsRNA agent. There are also described compositions aimed at serpin c1 gene, and methods using such agents for serpin c1 expression inhibition and treating individuals with haemorrhagic disorder such as haemophilia.

EFFECT: invention extends the range of agents for inhibiting serpin c1 expression.

184 cl, 31 dwg, 16 ex, 22 tbl