COMPOSITIONS FOR MODULATING C9ORF72 EXPRESSION Russian patent published in 2020 - IPC A61K31/711 C07H21/02 C12N15/11 C12N15/13 

FIELD: biotechnology.

SUBSTANCE: what is declared is a group of inventions comprising a modified oligonucleotide having the sequence SEQ ID NO: 33, or a salt thereof, the sodium salt of said oligonucleotide, a pharmaceutical composition, containing a therapeutically effective amount of said modified oligonucleotide for treating a disease associated with C9ORF72 (versions), a pharmaceutical composition, containing a therapeutically effective amount of a modified oligonucleotide sodium salt for treating a C9ORF72 associated disease. In one embodiment, the modified oligonucleotide is characterized by a nucleic acid sequence 5'-GCCCCTAGCGCGCGACTC-3' (SEQ ID NO: 33), wherein each cytosine is 5-methylcytosine; and where internucleoside linkages of the modified oligonucleotide, in direction 5'–3' are soosssssssssoooss, where each s is a thiophosphate bond, and each o is a phosphodiester linkage.

EFFECT: invention extends the range of products for treating diseases associated with C9ORF72.

20 cl, 17 tbl, 14 ex