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US 2026/0076990 A1Published application
Dsrna, use thereof and preparation method therefor
AssigneeTuojie Biotech (Shanghai) Co., Ltd.
InventorsYunfei Li, Yongyan Deng, Xiaoyan Lin, Yanhui Wang, Song Mao, Minyin Huang, Yaqin Zhou, Longfei Huang
Life of this patent
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Abstract
Provided are a dsRNA, and the use thereof and a preparation method therefor. Further provided are a pharmaceutical composition, a cell or a kit containing the dsRNA. The dsRNA can interfere with the expression of APOC3, and prevent and/or treat related diseases.
Claims23
A double-stranded ribonucleic acid (dsRNA) comprising a sense strand and an antisense strand that comprise contiguous nucleotides in a direction from the 5′ end to the 3′ end, wherein:
- the nucleotides at positions 7, 8, and 9 of the sense strand are 2′-fluoro-modified nucleotides, the nucleotide at position 5 is independently a 2′-methoxy-modified nucleotide or 2′-fluoro-modified nucleotide, and the nucleotides at the other positions are 2′-methoxy-modified nucleotides;
- the nucleotides at positions 2 and 14 of the antisense strand are 2′-fluoro-modified nucleotides, the nucleotides at positions 4, 6, 8, 9, 10, 12, 16, and 18 are independently 2′-methoxy-modified or 2′-fluoro-modified nucleotides, and the nucleotides at the other positions are 2′-methoxy-modified nucleotides;
- the number of 2′-fluoro-modified nucleotides in the antisense strand is 2-7;
- the antisense strand comprises a chemical modification represented by formula (I), a tautomer thereof, or a pharmaceutically acceptable salt thereof at nucleotide position 7:
- the chemical modification represented by formula (I) is selected from the group consisting of any one of the following structures:
- wherein B is the base of the nucleotide at position 7 of the 5′ region of the antisense strand when unmodified;
- and
- the dsRNA inhibits the expression of apolipoprotein C3 (APOC3).
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Dependencies are inferred from the claim text.