Hepatic fibrosis secondary to HCV infection can lead to cirrhosis and hepatic decompensation. Sustained virologic response (SVR) is possible with direct-acting antiviral drug regimens; however, patients with advanced fibrosis have an increased risk for HCC. Heat shock protein 47 (HSP47), a key collagen chaperone, has been implicated in fibrosis development. We evaluated the efficacy and safety of BMS-986263, a lipid nanoparticle delivering small interfering RNA designed to degrade HSP47 mRNA, for the treatment of advanced fibrosis.

中文翻译：

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BMS-986263 治疗晚期肝纤维化患者：随机、安慰剂对照 2 期试验的 36 周结果


HCV感染继发的肝纤维化可导致肝硬化和肝功能失代偿。直接作用的抗病毒药物治疗方案可以实现持续病毒学应答（SVR）；然而，晚期纤维化患者患 HCC 的风险增加。热休克蛋白 47 (HSP47) 是一种关键的胶原伴侣，与纤维化的发展有关。我们评估了 BMS-986263 的功效和安全性，BMS-986263 是一种脂质纳米颗粒，可传递小干扰 RNA，旨在降解HSP47 mRNA，用于治疗晚期纤维化。