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

NCTID	NCT05694598 (View at clinicaltrials.gov)
Description	A Multicenter, Open-Label, Non-Randomized, Uncontrolled Study of VGR-R01 in Patients with Bietti Crystalline Dystrophy. (Show More)
Indication	Bietti Crystalline Dystrophy
Compound Name	VGR-R01 (rAAV2/8-hCYP4V2)
Sponsor	Shanghai Vitalgen BioPharma Co., Ltd.
Funder Type	Industry
Status	Not yet recruiting
Enrollment Count	12

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
Dose 1	6.0 x 10^10 vg/eye
Dose 2	1.2 x 10^11 vg/eye
Dose 3	2.0 x 10^11 vg/eye
Dose 4	1.2 x 10^11 vg/eye (expansion dose)
Dose 5

No.of Trial Sites	1
Locations	China

Has US IND	False
Recent Updates	Phase I/II preliminary results presented at ASGCT 2024