ZVS101e in Patients With Bietti's Crystalline Dystrophy

Clinical Translation and Application Protocol for ZVS101e in the Gene Therapy of Bietti Crystalline Corneoretinal Dystrophy

The goal of this clinical trial is to evaluate the safety and early effectiveness of ZVS101e in patients with Bietti crystalline dystrophy who meet the eligibility criteria for treatment under the translational application program in the Hainan Boao Lecheng International Medical Tourism Pilot Zone.

Participants will:

undergo screening and baseline assessments to confirm eligibility; receive a single subretinal injection of ZVS101e in the study eye; complete follow-up visits over 4 weeks after treatment for safety monitoring and assessment of early effectiveness.

Inclusion Criteria:

1. Voluntarily participate in the research and sign the informed consent form, and be willing to complete the entire process according to the protocol requirements;
2. Clinically diagnosed with Bietti's Crystalline Dystrophy (BCD), age ≥ 18 years;
3. Confirmed by genetic testing to carry homozygous or compound heterozygous mutations in CYP4V2, and not complicated with other ophthalmic genetic diseases;
4. Target eye BCVA ≤ 60 ETDRS letters.

Exclusion Criteria:

Subjects will be excluded if they meet any 1 of the following exclusion criteria:

1. The target eye currently has or has a history of macular lesions; suffers from ocular diseases that may hinder surgery or interfere with the interpretation of study endpoints;
2. The target eye has previously undergone retinal reattachment surgery, vitrectomy, or any intraocular surgery within 3 months before enrollment;
3. Within 1 month before enrollment, suffered from viral infectious diseases that may affect the evaluation of the efficacy and safety of the investigational drug, or received antiviral vaccines;
4. The target eye has previously received gene therapy or stem cell therapy for BCD or other ocular diseases;
5. Known allergy to the drugs planned for use in the study;
6. The laboratory abnormalities are considered clinically significant;
7. Pregnant or lactating females.