A Phase I/II Clinical Study to Evaluate the Safety and Efficacy of VGN-R08b in Patients With Type III Gaucher's Disease

Inclusion criteria

• . The signing of the informed consent form should be completed when the subject is at least 2 years old but less than 18 years old.
• . The subject has a documented medical history of Gaucher disease confirmed by GCase enzyme activity testing, and has a double GBA1 gene mutation.
• . According to the investigator's assessment, the neurological signs and/or symptoms are consistent with type III Gaucher disease.
• . At the time of enrollment, the subject has horizontal eye movement disorders (including gaze paralysis, or delayed or absent saccades), but there is no severe motor dysfunction resulting in bedridden status.
• . The subject is currently receiving substrate reduction therapy (SRT) and/or high-dose ambroxol for Gaucher disease treatment. The subject is required to have been on stable treatment for at least 2 months before enrollment and the investigator determines that the treatment is ineffective for neurological symptoms, or is willing to discontinue the treatment at the time of enrollment (discontinuation 1 week before administration).
• . The subject is currently receiving and willing to continue stable peripheral treatment (including imiglucerase or other ERT, or SRT), and the peripheral symptoms of Gaucher disease are stable at the time of screening, that is, all of the following conditions are met: hemoglobin level ≥ 11.0 g/dL (female) or ≥ 12.0 g/dL (male), platelet count ≥ 100×109/L, spleen volume \< 10 times the normal value (MN), liver volume \< 1.5 MN, and no bone crisis or asymptomatic bone disease (such as bone necrosis and/or pathological fractures causing bone pain) within 3 months before screening.
• . (Applicable) Male and female subjects with reproductive potential must continue to use an effective contraceptive method (including abstinence) correctly from the screening period until at least 1 year after the start of treatment, and not donate sperm or eggs.