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

A Multicenter, Open-Label, Non-Randomized, Uncontrolled Study of VGR-R01 in Patients with Bietti Crystalline Dystrophy.

Key Inclusion Criteria:Able to provide informed consent and comply with requirements of the study;≥18 years and <70 years of age;Confirmed diagnosis of Bietti Crystalline Dystrophy and molecular diagnosis of CYP4V2 mutations (homozygotes or compound heterozygotes);BCVA ≤ 60 ETDRS letters in the study eye.Key Exclusion Criteria:Have insufficient viable retinal photoreceptor cells based on investigator's decision;Have current ocular or periocular infections, or endophthalmitis;Have any significant ocular disease/disorder other than BCD, including age-related macular degeneration, diabetic retinopathy, optic neuropathy, significant lens opacity, glaucoma, uveitis, retinal detachment, etc;Have intraocular surgery history except cataract surgery in the study eye;Have or potentially require of systemic medications that may cause eye injure;Have contraindications for corticosteroids or immunosuppressant;Unwilling or unable to have the planned follow-up;Abnormal coagulation function or other clinically significant abnormal laboratory results;Have malignancies or history of malignancies;History of immunodeficiency (acquired or congenital); Other protocol defined Inclusion/Exclusion criteria may apply.