A Clinical Study Evaluating LY-M001 Injection in the Treatment of Adult Patients with Type I Gaucher Disease

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 studyas well as possible adverse reactions, and sign the informed consent form (ICF)voluntarily.

3. Patients with confirmed double mutations in the GBA1 allele through laboratorytesting, and the glucocerebrosidase activity was reduced to less than 30% of thenormal value, and meeting the standard clinical diagnosis criteria for GD1.

4. Patients who meet a) or b) below:

5. Treated patients with Gaucher disease type I who had previously received enzymereplacement therapy (ERT) or substrate clearance therapy (SRT) with GD, were onstable medication, eluted 5 drugs for a half-life or more beforeadministration, or were comprehensively judged to be stable by theinvestigator.

6. Newly treated or untreated GD1 patients who meet one or more of the followingcriteria at screening:

• Hemoglobin ≥80g/L and less than the lower limit of normal;
• Platelets ≥40×109/L and less than the lower limit of normal;
• Hepatomegaly;
• Splenomegaly.

5. Negative pregnancy test for female subjects of childbearing potential (WOCBP).Notes: WOCBP is defined as the absence of postmenopausal status (continuousamenorrhea of at least 12 months with no identifiable cause other than menopause),and the absence of surgical (i.e., ovarian, salpingectomy, and/or hysterectomy) orInvestigator-determined cause of permanent infertility due to other causes (e.g.,lenticular hypoplasia) after menarche in female subjects.

6. The subject 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 theend of the study.

Exclusion Criteria:

1. AAV8 neutralizing antibody positive.

2. Patients with clinically diagnosed Gaucher disease type II or III (GD2 or 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 GD as judged by the Investigator.

5. Treatment or disposal of investigational drugs or investigational devices receivedin other clinical studies within 28 days prior to screening or within 5 half-lives (drugs only), whichever is older.

6. Evidence of clinically significant liver disease, fragile liver, or history ofexposure to hepatotoxins that meets, but is not limited to, any of the following atthe time of screening:

• Progressive hepatomegaly larger than 3 times the normal volume.

• History of stage 2 or above liver fibrosis.

• AST, ALT, or TBIL are 1.5 times higher than ULN.

• A history of alcohol or drug abuse within the previous 2 years (defined ashaving consumed more than 14 standard units of alcohol per week [1 standardunit containing 14 g of alcohol, such as 360 mL beer, 45 mL spirits containing 40% or more alcohol, or 150 mL wine]).

• Hepatitis B surface antigen (HBsAg) positive and HBV deoxyribonucleic acid (HBV-DNA) positive (HBV-DNA>103 copy number /mL); Or take hepatitis B drugs (such as interferon, lamivudine, adefovir and entecavir); Or antibodies tohepatitis C virus (HCV) and positive for hepatitis C virus RNA.

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

8. Severe hyperlipidemia (triglycerides > 11.29mol/L).

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

10. 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.

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

12. Clinically diagnosed or investigator-determined serious cardiovascular disease (suchas heart failure ≥3 from the New York College of Cardiology [NYHA]).

13. Hypersensitivity to any component of LY-M001 injection.

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

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

16. History of cancer within 5 years prior to screening, or currently active neoplasticdisease, except for basal or squamous cell carcinoma of the skin or carcinoma insitu that has been definitively treated.

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

18. Other conditions that, in the opinion of the Investigator, make the subjectunsuitable for the study.