MODULATORS OF COMPLEMENT FACTOR B Russian patent published in 2018 - IPC A61K48/00 C12N15/113 

FIELD: biochemistry.

SUBSTANCE: invention relates to biochemistry. Described is a compound capable of inhibiting complement factor B (CFB) expression, containing a modified oligonucleotide consisting of 10–30 linked nucleosides, having a nucleotide base sequence that contains any of SEQ ID NO: 440, 198, 228, 237, 444, 448, 450, 453, 455, 549 and 598, wherein said modified oligonucleotide comprises: gap segment consisting of linked deoxynucleosides; wing segment 5' consisting of linked nucleosides; and wing segment 3' consisting of linked nucleosides, wherein said gap segment is located between wing segment 5' and wing segment 3', and each nucleoside of each wing segment contains modified sugar. Provided is a composition comprising said compound and a method of treatment by using said compound.

EFFECT: invention provides a wider range of agents for treating, preventing, or ameliorating a disease associated with dysregulation of the complement alternative pathway by administering a complement factor B (CFB) specific inhibitor to a subject.

22 cl, 106 tbl, 20 ex