A Study of the Safety and Preliminary Efficacy of LY-M001 Injection in the Treatment of Adult Patients with Gaucher Disease Type I

Inclusion Criteria:

1. Age ≥ 18 years and ≤ 60 years, male or female.

2. The subjects should fully understand the purpose, nature and method of this study aswell as possible adverse reactions, and sign the informed consent form (ICF)voluntarily.

3. Patients with GD1 who have confirmed double mutations in the Gba1 allele bylaboratory testing and meet the standard for clinical diagnosis of Gaucher disease (i.e., reduced GCase enzyme activity to less than 30% of normal values).

4. The subjects were type I patients with Gaucher disease. Patients with type I Gaucherdisease who had received specific treatment in the past required 5 half-lives ofelution

5. Negative pregnancy test for female subjects of childbearing potential 6.6.Thesubject and his/her partner have no plans to have children during the screeningperiod and within 6 months after the end of the study, and voluntarily takeeffective contraceptive measures (such as abstinence, condom, etc.); and the subjecthad no plans to donate sperm or eggs.

7.Subjects are not to donate blood during the study and for at least 1 year after the end of the study.

Exclusion Criteria:

1. AAV8 neutralizing antibody is strongly positive.

2. Patients with clinically suspected Gaucher disease type II (GD2) or Gaucher diseasetype III (GD3).

3. Active and progressive bone disease that is expected to require surgical treatmentwithin the next 6 months.

4. Subject has idiopathic thrombocytopenic purpura (ITP), thrombotic thrombocytopenicpurpura (TTP), thrombocytopenia, anemia, hepatomegaly, splenomegaly, and/orosteoporosis unrelated to Gaucher disease as judged by the investigator.

5. Treatment or disposition of an investigational drug or investigational device foranother clinical investigation within 28 days or 5 half-lives (only for drugs),whichever is longer, prior to Screening.

6. Evidence of clinically significant liver disease, fragile liver, or history ofhepatotoxin exposure, meeting at screening, but not limited to, any of thefollowing:

• Progressive hepatomegaly and greater than 3 times normal volume.

• History of stage 2 or greater liver fibrosis.

• AST, ALT or TBIL greater than 1.5 times the upper limit of normal (ULN).

• History of alcohol or drug abuse within the previous 2 years.

• Hepatitis B surface antigen (HBsAg) positive, and hepatitis B virus DNApositive (HBV-DNA > 103 copies/mL); or taking hepatitis B virus drugs (such asinterferon, lamivudine, adefovir and entecavir); or hepatitis C virus (HCV)antibody positive.

7. Human immunodeficiency virus (HIV) antibody positive or Treponema pallidum antibodypositive.

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

9. Uncontrolled concomitant disease or infectious disease (need to be judged by theinvestigator based on clinical practice).

10. Subject had undergone splenectomy and were scheduled to undergo splenectomy duringthe study period.

11. Karnofsky score (KPS) < 70.

12. The subject has received or plans to receive bone marrow transplantation,hematopoietic stem cell transplantation and/or major organ transplantation,including but not limited to liver transplantation, kidney transplantation, etc.

13. Subject has received erythropoietin, transfusion, or red blood cell transfusionwithin 3 months prior to screening ;or platelet transfusion within 1 month prior toscreening.

14. Clinically diagnosed or significant cardiovascular disease as judged by theinvestigator (e.g., New York Heart Association [NYHA] class ≥ 3 heart failure).

15. Hypersensitivity to any component of LY-M001 Injection.

16. Previous treatment with any type of gene therapy or cell therapy.

17. Use of systemic immunosuppressive agents or steroid therapy other than thoserequired by the protocol for prophylactic administration within 3 months prior todosing.

18. History of cancer within 5 years of screening, except for completely resectednon-melanoma skin cancer, non-metastatic prostate cancer, and completely treatedductal carcinoma in situ.

19. Has received a live attenuated vaccine within 4 months prior to screening or plansto receive a live attenuated vaccine during the clinical trial.

20. Other conditions that, in the opinion of the investigator, make the subjectunsuitable for the study.