Wilson's disease (WD), also known as Wilson's disease, is a rare autosomal recessive metabolic disorder caused by a mutation of the copper transport ATPase β (ATP7B) gene located on the long arm of chromosome 13 (13q14.3). This leads to accumulation of copper ions in multiple organs such as liver, brain and kidney, resulting in organ involvement. In this study, LY-M003 Injection is a gene therapy products with rAAV8 vector. After a single intravenous infusion, LY-M003 can be transduced to the target organ of liver and express the ATP7B in hepatocytese.
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Incidence of adverse events (AEs) and serious adverse events (SAEs) within 52 weeks after the injection of LY-M003
Timeframe: From enrollment to 52 weeks after administration
Incidence of dose-limiting toxicity (DLT) events assessed within at least 28 days following LY-M003 infusion
Timeframe: From enrollment to 52 weeks after administration