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

A Phase 1 Study to Assess the Safety and Tolerability of VGR-R01 in Patients With Bietti Crystalline Dystrophy

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

VGR-R01 is a novel AAV vector carrying the human CYP4V2 coding sequence. This study is intended to evaluate the safety and tolerability of a single subretinal administration of VGR-R01. All subjects will undergo at least 52 weeks of safety observation and will be encouraged to enroll in an extension study to evaluate the long-term safety of VGR-R01 for a total of five years.

Key Inclusion Criteria:

1. Able to provide informed consent and comply with requirements of the study;
2. ≥18 years and <70 years of age;
3. Confirmed diagnosis of Bietti Crystalline Dystrophy and molecular diagnosis of CYP4V2 mutations (homozygotes or compound heterozygotes);
4. BCVA ≤ 60 ETDRS letters in the study eye.

Key Exclusion Criteria:

1. Have insufficient viable retinal photoreceptor cells based on investigator's decision;
2. Have current ocular or periocular infections, or endophthalmitis;
3. 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;
4. Have intraocular surgery history except cataract surgery in the study eye;
5. Have or potentially require of systemic medications that may cause eye injure;
6. Have contraindications for corticosteroids or immunosuppressant;
7. Unwilling or unable to have the planned follow-up;
8. Abnormal coagulation function or other clinically significant abnormal laboratory results;
9. Have malignancies or history of malignancies;
10. History of immunodeficiency (acquired or congenital); Other protocol defined Inclusion/Exclusion criteria may apply.