Safety and Efficacy Study of NGGT001 in Bietti Crystalline Corneoretinal Dystrophy

NCTID	NCT06302608 (View at clinicaltrials.gov)
Description	Evaluate the safety and tolerability of NGGT001 subretinal injection for the treatment of crystalline retinal degeneration (BCD) (Show More)
Indication	Bietti Crystalline Dystrophy
Compound Name	NGGT001
Sponsor	Xiamen Ophthalmology Center Affiliated to Xiamen University
Funder Type	Other
Status	Active not recruiting
Enrollment Count	6

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
Editor Type
Dose 1	1.5 x 10^11 vg/eye
Dose 2	3.0 x 10^11 vg/eye
Dose 3
Dose 4
Dose 5

No.of Trial Sites	1
Locations	China

Has US IND	False
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