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Gene Therapy Trial Report

Summary

Efficacy and Safety of ZVS101e in Patients With Bietti 's Crystalline Dystrophy


NCTID NCT06743646 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Bietti Crystalline Dystrophy
Disease Ontology Term DOID:0050664
Drug Product Name ZVS101e
Drug Product Alias puliretgene parvec
Drug Product Description rAAV2/8-hCYP4V2
Sponsor Chigenovo Co., Ltd
Funder Type Network
Recruitment Status πŸ”„
Active not recruiting
Enrollment Count πŸ”„ 63 (ACTUAL)
Results Posted Not Available

Therapy Information


Target Gene/Variant CYP4V2
Regulatory Element CAG (promoter)
Therapeutic Modality Gene transfer
Therapy Route in vivo
Mechanism of Action Functional gene replacement
Route of Administration Subretinal
Drug Product Type Viral vector
Gene Delivery System Type Viral transduction
Vector Type πŸ”„ Adeno-associated virus
Viral Vector Subtype AAV2/8
Dose 1 πŸ”„ IIT trial (NCT0422107): 7.5E10 vg/eye (n=11)
Dose 2 πŸ”„ IIT trial (NCT05714904): 7.5E10 vg/eye (n=6)
Dose 3 πŸ”„ Phase 1/2 (NCT05832684): 7.5E10 vg/eye (n=3), 1.5E11 vg/eye (n=3), 2.2E11 vg/eye (n=1)
Dose 4 πŸ”„ Phase 3: 7.5E10 vg/eye (Expansion/pivotal dose)

Study Record Dates


Current Phase Phase3
Submit Date 2024-12-17
Completion Date 2030-06-30
Last Update πŸ”„ 2026-10-29

Participation Criteria


Eligible Age >=18 Years
Standard Ages Adult, Older adult
Sexes Eligible for Study ALL
Eligibility Criteria
Inclusion Criteria: 1. All of the following inclusion criteria should be met for enrollment into the trial: 2. Fully understand the purpose and requirements of this trial, voluntarily participate in the clinical trial and sign the informed consent form, and be able to complete all trial procedures as required by the protocol; 3. Clinical diagnosis of Bietti's crystalline dystrophy (BCD), age≥18 years ; 4. Genetic testing confirmed biallelic CYP4V2 mutations without other ophthalmic genetic diseases; 5. Best-corrected visual acuity of 5-60 ETDRS letters. Exclusion Criteria: 1. The study eye has or has had macular lesions such as macular hole or macular neovascularization; glaucoma, diabetic retinopathy, or any other ocular disease that may preclude surgery or interfere with interpretation of the study endpoints 2. The study eye had received the following intraocular surgical treatments: retinal reattachment, vitrectomy; 3. The study eye had received any intraocular surgery, such as phacoemulsification 3 months prior to enrollment; 4. Previously treatment of either eye with gene therapy or stem cell therapy for BCD and other ocular diseases, including but not limited to viral vector gene therapy, RNA therapy; 5. Pregnant or lactating women;
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites πŸ”„ 5
Locations China

Regulatory Information


Has US IND False
FDA Designations πŸ”„ Orphan Drug Designation, Regenerative Medicine Advanced Therapy (RMAT)
Recent Updates Marketing application submitted in 2026, primary market is in China

Resources/Links