A Clinical Study to Evaluate the Safety and Efficacy of LY-M003 Injection in Patients With Wilson Disease

Inclusion Criteria:

1. The subject must be able to fully understood the purpose, nature, method, andpossible adverse effects of the study, must be able to voluntarily participate inthe study and voluntarily able to provide the written informed consent form (ICF).

2. Patients diagnosed with Wilson Disease .

3. Wilson Disease (WD) patients confirmed by laboratory tests to have biallelicmutations in the ATP7B gene.

4. 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 screeningperiod.

5. Subjects must restrict food with high copper content for at least 6 months prior toscreening and continue this restriction during the entire duration of studyparticipation.

6. Subjects must be willing to refrain from donating blood, organs, tissues or cellsduring study participation.

7. Negative pregnancy test in women of childbearing potential (WOCBP).

8. Subjects and their partners who have no childbearing plans from the screening periodto 6 months after the end of the study and are willing to adopt effectivecontraceptive measures (e.g., abstinence, condoms, etc.); subjects have no plans todonate sperm or ova.

Exclusion Criteria:

1. AAV8 neutralizing antibody titer > 1:10 .

2. Active gastrointestinal bleeding within the past 3 months.

3. Decompensated cirrhosis or advanced hepatic disease, manifested as portalhypertension, ascites, splenomegaly, esophageal varices, hepatic encephalopathy,etc.

4. Subjects with other liver diseases as determined by the investigator, such as immunehepatitis, alcoholic liver disease, primary biliary cholangitis, primary sclerosingcholangitis, and/or drug or toxic liver disease

5. Subjects considered as complicated with severe hypersplenism and requiringsplenectomy as judged by the investigator.

6. Model for End-Stage Liver Disease (MELD) Score > 13.

7. Other disorders of copper metabolism, such as chronic cholestatic liver diseases,disorders of glycosylation, copper metabolism disorders, etc.

8. History of noncompliance with copper chelators or zinc agents within 6 months priorto screening, as determined by the investigator.

9. Subjects with treatment-experienced WD who have ALT and/or AST 5 times greater thanthe upper limit of normal (ULN).

10. Severe central nervous system symptoms urgent for intensive hospitalization judgedby the investigator.

11. Hemoglobin < 90 g/L.

12. A history of epileptic seizures or other diseases that may potentially affectcompliance with study procedures within 6 months prior to the screening period.

13. Hepatitis B surface antigen (HBsAg) positive, hepatitis C virus (HCV) antibodypositive, human immunodeficiency virus (HIV) antibody positive or Treponema pallidumantibody positive.

14. Subjects with end-stage renal disease receiving dialysis (chronic kidney diseasestage 3 and above) or creatinine clearance < 60 mL/min.

15. Severe hyperlipidemia (triglycerides > 1000 mg/dL).

16. Subject received or plans to receive bone marrow transplantation, hematopoietic stemcell transplantation and/or major organ transplantation, including but not limitedto liver transplantation, kidney transplantation, etc.

17. Clinically diagnosed or judged as serious cardiovascular disease by the investigator (eg, classification of heart failure ≥ 3 according to New York Heart Association [NYHA]).

18. Patients with uncontrolled concomitant diseases or infectious diseases as judged bythe investigator.

19. Subjects who have hypersensitivity to any component of LY-M003 injection.

20. Subjects who have previously received gene therapy or cell therapy of any kind.

21. Subjects who use systemic immunosuppressive agents or receive steroid therapy within 3 months prior to dosing (except for prophylactic immunosuppressive therapy asspecified in protocol).

22. Subjects with history of cancer within 5 years prior to screening, except forcompletely resected non-melanoma skin cancer, non-metastatic prostate cancer andcompletely cured ductal carcinoma in situ.

23. Subjects who have vaccinated with attenuated live vaccine within 4 months prior toscreening or plan to receive a live attenuated vaccine during the clinical trial.

24. Subjects who have received treatment or disposition with another investigationaldrug or investigational device within 28 days or 5 half-lives (drug only), whicheveris longer, prior to screening.

25. Pregnant women (or women planning to become pregnant) or lactating women.

26. Other circumstances in which the investigator deems the subject inappropriate forstudy participation.