COMPOUND FOR INHIBITING C3 GENE EXPRESSION, PHARMACEUTICAL COMPOSITION AND USE THEREOF

Abstract: a compound for inhibiting c3 gene expression, a pharmaceutical composition and a use thereof, belonging to the technical field of small nucleic acid drug delivery. a compound conjugated with an oligonucleotide and a pharmaceutical composition thereof can significantly inhibit the expression of c3 mrna in animal liver tissue and c3 protein in serum and can reduce c3 deposition in renal tissue and improve the course of disease featuring c3 deposition in renal tissue. the no-adverse-effect level (noael) in sd rats and cynomolgus monkeys is 300 mg/kg. the compound conjugated with a oligonucleotide and the pharmaceutical composition thereof are helpful in alleviating, preventing and/or treating diseases or conditions mediated by dysregulated c3 gene expression.

Inventor(s): Yuanyu HUANG, Haitao LI, Lina KONG, Zhibin FAN, Yongxin GAO, Xiaofeng HAN

CPC Classification: A61K47/549 ({Sugars, nucleosides, nucleotides or nucleic acids})

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