BEBT-730

BEBT-730 is a kidney-targeted small interfering RNA (siRNA) therapeutic developed by Guangzhou BeBetter Medicine Technology for the treatment of hyperuricemia and gout. Utilizing the company's proprietary Peptide-Oligonucleotide Conjugates (POC) platform, BEBT-730 selectively delivers siRNA to proximal tubule epithelial cells (PTECs) in the kidney. Its mechanism involves the downregulation of the Urate Transporter 1 (URAT1) mRNA and protein, which is the primary protein responsible for uric acid reabsorption in the renal tubules. By reducing URAT1 levels, the drug promotes uric acid excretion and lowers serum urate levels. Preclinical studies have demonstrated significant URAT1 mRNA knockdown (up to 93%) and improvements in renal function markers without evidence of systemic toxicity in mouse models.