NUCLEIC ACID, PHARMACEUTICAL COMPOSITION AND CONJUGATE, METHOD OF PRODUCTION AND USE Russian patent published in 2024 - IPC A61K48/00 A61K47/54 A61K47/26 C12N15/113 A61P21/04 A61P37/00 

FIELD: biotechnology.

SUBSTANCE: disclosed is a siRNA conjugate having a structure represented by formula (308), where L₁, M₁, R₂, R₃, R₁₀-R₁₅, m₁-m₃, n₁-n₃ are defined in the claims, and which siRNA is capable of inhibiting the expression of complement protein 5 (C5) gene, where each nucleotide in siRNA is an independent modified or unmodified nucleotide. siRNA contains a sense strand and an antisense strand. Sense strand contains a nucleotide sequence I, and the nucleotide sequence I has the same length and not more than three nucleotide differences from the nucleotide sequence shown in SEQ ID NO: 1. Antisense strand contains a nucleotide sequence II, and the nucleotide sequence II has the same length and not more than three nucleotide differences from the nucleotide sequence shown in SEQ ID NO: 2.

EFFECT: what is presented is using the siRNA conjugate for producing a drug for treating and / or preventing myasthenia gravis and a method for inhibiting expression of the C5 gene in hepatocyte.

24 cl, 2 dwg, 6 tbl, 4 ex