Sirna for inhibiting expression of urate transporter 1 gene, and drug and use thereof
By designing siRNA that specifically targets the URAT1 gene, the problems of side effects, tolerability, and high dosing frequency of existing URAT1 small molecule inhibitors have been solved, achieving efficient and safe treatment for hyperuricemia.
WO2026037289A1 Publication Date: 2026-02-19BEBETTER MED INC
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Patent Information
- Application Number
- PCT/CN2025/114129
- Authority / Receiving Office
- WO · WO
- Patent Type
- Applications
- Current Assignee / Owner
- Priority Date
- 2025-04-03
- Filing Date
- 2025-08-12
- Publication Date
- 2026-02-19
AI Technical Summary
Technical Problem
Existing URAT1 small molecule inhibitors have problems such as side effects, drug tolerance, insufficient specificity, and high dosing frequency when treating hyperuricemia, which affect treatment efficacy and patient compliance.
Method used
By using siRNA that specifically targets the URAT1 gene, and through the design of specific nucleotide sequences and modification methods, double-stranded siRNA is formed to inhibit URAT1 gene expression and reduce uric acid levels.
Benefits of technology
It improves the specificity and safety of treatment, reduces side effects, lowers the frequency of administration, and improves patient compliance.
✦ Generated by Eureka AI based on patent content.
Abstract
Disclosed in the present invention is an siRNA for inhibiting the expression of a urate transporter 1 (URAT1) gene in human cells. In the present invention, a plurality of siRNAs capable of inhibiting URAT1 expression are obtained by screening, and on this basis, appropriate modifications are performed to obtain sequences with improved target silencing ability. Drugs with the siRNAs as active ingredients are expected to be clinically applicable in the prevention and treatment of diseases related to the URAT1 target, such as gout, hyperuricemia, gouty arthritis and renal calculi.
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