Study to Evaluate the Efficacy and Safety of VGR-R01 Gene Therapy in Patients with Bietti Crystalline Dystrophy

NCTID	NCT06699108 (View at clinicaltrials.gov)
Description	This is a phase 3 study to evaluate the efficacy and safety of VGR-R01 in subjects with Bietti Crystalline Dystrophy. This is a multicenter, randomized controlled study which will enroll 45 subjects. (Show More)
Development Status	Active
Indication	Bietti Crystalline Dystrophy
Disease Ontology Term	DOID:0050664
Compound Name	VGR-R01
Compound Alias	rAAV2/8-hCYP4V2
Sponsor	Shanghai Vitalgen BioPharma Co., Ltd.
Funder Type	Industry
Recruitment Status	Not yet recruiting
Enrollment Count	45 (ESTIMATED)
Results Posted	Not Available

Target Gene/Variant	CYP4V2
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Subretinal
Drug Product Type	Viral vector
Target Tissue/Cell
Delivery System	Viral transduction
Vector Type	AAV2/8
Editor Type	none
Dose 1	6.0E10 vg/eye
Dose 2	1.2E11 vg/eye (expansion dose)
Dose 3	2.0E11 vg/eye
Dose 4
Dose 5

No.of Trial Sites	1
Locations	China

Has US IND	False
FDA Designations	Orphan Drug Designation
Recent Updates	Phase 3 enrollment complete, BLA submission target 2H 2026