ZVS101e in Patients With Bietti's Crystalline Dystrophy (ZVS101e-BCD)

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.

Study Overview

• Status: Not yet recruiting
• Conditions: Bietti's Crystalline Dystrophy; Bietti Crystalline Corneoretinal Dystrophy
• Intervention / Treatment: Drug: ZVS101e

• Study Type: Interventional
• Enrollment (Estimated): 300
• Phase: Not Applicable

Contacts and Locations

• Study Contact: Name: Jinlu Zhang, MD; Phone Number: +86-15810570898; Email: zhangjinlu@chinagene.cc
• Study Contact Backup: Name: Tingting Wu, BS; Phone Number: +86-18500191916; Email: wutingting@chinagene.cc

Study Locations

• China
  • Hainan
    • Qionghai, Hainan, China
      • Boao Super Hospital
    • Qionghai, Hainan, China
      • Hainan Boao Ophthalmology Center, Zhongshan Ophthalmic Center, Sun Yat-sen University

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: ZVS101e Treatment Arm	Drug: ZVS101e; ZVS101e injection contains a recombinant adeno-associated virus serotype 8 (rAAV8) vector that expresses human CYP4V2 protein.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Change from baseline in Best corrected visual acuity (BCVA)	From enrollment to the end of treatment at 52 weeks
The incidence and severity of adverse events and serious adverse events	From enrollment to the end of treatment at 52 weeks

Collaborators and Investigators

• Sponsor: Chigenovo Co., Ltd

Publications and helpful links

General Publications

• Jia R, Meng X, Chen S, Zhang F, Du J, Liu X, Yang L. AAV-mediated gene-replacement therapy restores viability of BCD patient iPSC derived RPE cells and vision of Cyp4v3 knockout mice. Hum Mol Genet. 2023 Jan 1;32(1):122-138. doi: 10.1093/hmg/ddac181.
• Wang J, Zhang J, Yu S, Li H, Chen S, Luo J, Wang H, Guan Y, Zhang H, Yin S, Wang H, Li H, Liu J, Zhu J, Yang Q, Sha Y, Zhang C, Yang Y, Yang X, Zhang X, Zhao X, Wang L, Yang L, Wei W. Gene replacement therapy in Bietti crystalline corneoretinal dystrophy: an open-label, single-arm, exploratory trial. Signal Transduct Target Ther. 2024 Apr 24;9(1):95. doi: 10.1038/s41392-024-01806-3.

Study record dates

Study Major Dates

• Study Start (Estimated): July 30, 2026
• Primary Completion (Estimated): December 30, 2030
• Study Completion (Estimated): December 1, 2035

Study Registration Dates

• First Submitted: June 12, 2026
• First Submitted That Met QC Criteria: June 12, 2026
• First Posted (Actual): June 17, 2026

Study Record Updates

• Last Update Posted (Actual): June 17, 2026
• Last Update Submitted That Met QC Criteria: June 12, 2026
• Last Verified: June 1, 2026

More Information

Terms related to this study

• Keywords: Bietti Crystalline Corneoretinal Dystrophy; ZVS101e; AAV8-hCYP4V2; Bietti's Crystalline Dystrophy; gene replacement treatment
• Additional Relevant MeSH Terms: Bietti Crystalline Dystrophy
• Other Study ID Numbers: ZYA-2025-003

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No