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)
Development Status	Active
Indication	Bietti Crystalline Dystrophy
Disease Ontology Term	DOID:0050664
Compound Name	NGGT001
Sponsor	Xiamen Ophthalmology Center Affiliated to Xiamen University
Funder Type	Other
Recruitment Status	Active not recruiting
Enrollment Count	6
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
Editor Type	none
Dose 1	1.5E11 vg/eye
Dose 2	3.0E11 vg/eye
Dose 3
Dose 4
Dose 5

No.of Trial Sites	1
Locations	China