A Prospective, Single-center, Open, Single-arm, Single-dose Clinical Study Evaluating the Safety, Tolerability, and Efficacy of LY-M003 Injection in Adults with Wilson Disease
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, rAAV8 as the carrier of gene therapy products. After a single intravenous infusion, ATP7B protein can be specifically transduced to the target organ liver and expressed in hepatocytes for a long time.
• The subject must be able to fully understood the purpose, nature, method, and possible adverse effects of the study, must be able to voluntarily participate in the study and voluntarily able to provide the written informed consent form (ICF).
• Subjects with confirmed Wilson Disease: Leipzig scoring system assessment ≥ 4 points.
• Subjects with confirmed WD bichromosomal ATP7B gene mutation or missing by laboratory testing.
• Subjects must be treatment-experienced to WD who have received standard treatment (eg, D-penicillamine or zinc acetate) for at least 6 months prior to the screening period.
• Subjects must restrict food with high copper content for at least 6 months prior to screening and continue this restriction during the entire duration of study participation.
• Subjects must be willing to refrain from donating blood, organs, tissues or cells during study participation.
• Negative pregnancy test in women of childbearing potential (WOCBP).