CA3140233A1 — Nucleic acid, pharmaceutical composition and conjugate, preparation method and use

USPTO Abstract

Provided are an siRNA for inhibiting the expression of complement protein 5 (C5) gene, and a pharmaceutical composition and conjugate containing the siRNA. Each nucleotide in the siRNA is independently a modified or unmodified nucleotide. The siRNA comprises a sense strand and an antisense strand. The sense strand comprises nucleotide sequence I, and the nucleotide sequence I has the same length as that of the nucleotide sequence set forth in SEQ ID NO: 1 and has no more than 3 nucleotide differences. The antisense strand comprises nucleotide sequence II, and the nucleotide sequence II has the same length as that of the nucleotide sequence set forth in SEQ ID NO: 2 and no more than 3 nucleotide differences. Also provided is a method for using the siRNA and the pharmaceutical composition and conjugate thereof in the treatment and/or prevention of complement-mediated related diseases such as myasthenia gravis (MG).