Safety and Tolerability of VGR-R01 for Patients With Bietti Crystalline Dystrophy

Incidence of adverse events

An adverse event (AE) is any untoward medical occurrence in a clinical investigation participant administered a product; the event will not need to have a causal relationship with the treatment.

Time frame: Baseline up to Week 52

Incidence of serious adverse events

A serious adverse event (SAE) is any untoward medical occurrence at any dose that resulted in death; life threatening; require inpatient hospitalization or prolongation of existing hospitalization; result in persistent or significant disability/incapacity; result in congenital anomaly/birth defect.

Time frame: Baseline up to Week 52

Number of Participants with Clinically Significant Change from Baseline in Vital Signs

Vital signs (temperature, respiratory rate, pulse rate, systolic and diastolic blood pressure) will be obtained with participant in the seated position, after having sat calmly for at least 5 minutes. Clinical significance of vital signs will be determined at the investigator's discretion.

Time frame: Baseline up to Week 52

Number of Participants with Clinically Laboratory Abnormalities

Laboratory Tests will include hematology, coagulation, blood chemistry, urinalysis, serology, and pregnancy test, etc. If any potential changes accompanied by clinical symptoms, or results in a change of medical intervention, the findings will be considered as clinically significant based on investigator's decision.

Time frame: Baseline up to Week 52

Number of Participants with Clinically Significant Change from Baseline in Ophthalmic Examination Findings

Ophthalmic Examination will include BCVA, IOP, slitlamp examination, angiography and SD-OCT, etc. If any potential changes accompanied by clinical symptoms, or results in a change of medical intervention, the findings will be considered as clinically significant based on investigator's decision.

Time frame: Baseline up to Week 52