COMPOSITIONS FOR MODULATING TAU PROTEIN EXPRESSION Russian patent published in 2020 - IPC A61K48/00 C07H21/04 

FIELD: biotechnology.

SUBSTANCE: described is a modified oligonucleotide or salt thereof, consisting of 12–50 bound nucleosides and having a sequence of nitrogen bases, containing at least 8 consecutive nitrogen bases according to sequence of nitrogen bases from SEQ ID NO: 1223 or SEQ ID NO: 1634, wherein the nitrogen base sequence of the modified oligonucleotide is at least 90 % complementary to SEQ ID NO: 1 for reducing Tau expression. Also described is a modified oligonucleotide or salt thereof, consisting of 12–50 bound nucleosides and containing a sequence of nitrogen bases, containing at least 8, at least 9, at least 10, at least 11, at least 12, at least 13, at least 14, at least 15, at least 16, at least 17 , at least 18, at least 19, or at least 20 consecutive nitrogen bases, complementary to fragment of nitrogen bases equivalent to length 120043–120077 from SEQ ID No: 1 for reducing Tau expression. Also disclosed is a single-chain modified oligonucleotide or its salt consisting of 18 bound nucleosides and having a sequence of nitrogen bases containing 18 adjacent nitrogen bases in sequence of SEQ ID NO: 1634. Also disclosed is a single-chain modified oligonucleotide or its salt consisting of 18 bound nucleosides and having a sequence of nitrogen bases, containing 18 adjacent nitrogen bases in sequence of SEQ ID NO: 1223. Also provided is a composition for treating a neurodegenerative disease, disorder or condition, containing described modified oligonucleotide or its salt or presented single-chain modified oligonucleotide or its salt and at least one of pharmaceutically acceptable carrier or diluent.

EFFECT: invention reduces expression of mRNA and tau protein; use of the compound and composition is useful for treating, preventing or improving tau-accompanying diseases, disorders and conditions.

31 cl, 68 tbl, 20 ex