BEBT-730
BEBT-730 is specifically designed to deliver URAT1-targeting siRNA to PTECs, where URAT1 plays a central role in uric acid reabsorption. It reduces URAT1 mRNA in the kidney cells up to 93% reduction at 10 nM and downregulates URAT1 protein in PTECs, improving uric acid levels and kidney functions in a diseased mouse model. No changes in body weight, food intake, liver, or kidney function markers are observed after repeat doses in non-GLP toxicity study in CD1 mice.
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