Safety and Efficacy Study of NGGT001 in Bietti Crystalline Corneoretinal Dystrophy Subjects

NCTID	NCT06706427 (View at clinicaltrials.gov)
Description	The objective of this study is to evaluate the safety, tolerability, and efficacy of subretinal injection of NGGT001 in patients with Bietti Crystalline Corneoretinal Dystrophy (BCD) and to recommend the optimal dosage for future clinical administration. (Show More)
Development Status	Active
Indication	Bietti Crystalline Dystrophy
Disease Ontology Term	DOID:0050664
Compound Name	NGGT001
Sponsor	NGGT (Suzhou) Biotechnology Co., Ltd.
Funder Type	Industry
Recruitment Status	Active not recruiting
Enrollment Count	12
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	2
Locations	China