METHODS AND COMPOSITIONS FOR TREATMENT OF SERPINC1-ASSOCIATED DISORDER Russian patent published in 2021 - IPC A61K31/713 C12N15/113 C07H21/04 A61P7/04 

FIELD: medicine; hematology.

SUBSTANCE: group of inventions relates to the field of medicine, namely to hematology and hereditary diseases, it is intended for the treatment of subjects with Serpinc1-associated diseases. To prevent at least one symptom of hemophilia in a subject who has hemophilia A or hemophilia B, a dose of 50 mg to 90 mg of a double-stranded ribonucleic acid (hereinafter – dsRNA) molecule is injected subcutaneously to a subject who needs it once a month. The dsRNA molecule includes a sense chain and an antisense strand, where the sense strand contains the sequence 5’-GfsgsUfuAfaCfaCfCfAfuUfuAfcUfuCfaAf-3’ (SEQ ID NO:13) and the antisense strand contains the sequence 5’-usUfsgAfaGfuAfaAfuggUfgUfuAfaCfcsasg-3’ (SEQ ID NO:14), where a, c, g and u are 2'-O-methyl (2'-OMe) A, C, G and U, respectively; Af, Cf, Gf and Uf are 2'-fluorine A, C, G and U, respectively; and s is a phosphorothioate bond. A method for treating a subject who has hemophilia A or hemophilia B; a method for reducing the frequency of bleeding episodes in a subject who has hemophilia A or hemophilia B; a method for reducing the annual bleeding rate (ABR) in a subject who has hemophilia A or hemophilia B; and a method for reducing the annual spontaneous bleeding rate (AsBR) in a subject who has hemophilia A or hemophilia B are also proposed. In other embodiments, alternative options of the implementation of these methods and a set for the implementation of these methods are provided.

EFFECT: use of the group of inventions makes it possible to increase the effectiveness of treatment of subjects with Serpinc1-associated diseases, due to the extremely effective suppression of Serpinc1 expression and the exceptional duration of suppression of Serpinc1 expression.

40 cl, 20 dwg, 3 tbl, 3 ex