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SGB-3403 is an investigational small interfering RNA (siRNA) drug developed by Sanegene Bio for the treatment of hypercholesterolemia. It is a GalNAc-conjugated siRNA that specifically targets proprotein convertase subtilisin/kexin type 9 (PCSK9), a key regulator of low-density lipoprotein cholesterol (LDL-C) metabolism. By inhibiting PCSK9 synthesis in the liver through RNA interference, SGB-3403 reduces circulating LDL-C levels. Preclinical studies have demonstrated good safety and tolerability, as well as superior efficacy compared to benchmark products, with LDL-C reductions of up to 40%–70% at lower doses and less frequent administration. The drug utilizes Sanegene's proprietary GalNAc platform technology for targeted delivery to hepatocytes. As of 2024, SGB-3403 is in Phase 1 clinical trials in Australia and China for hypercholesterolemia[1][2][4].
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