Safety and Efficacy of a Single Subretinal Injection of JWK006 Gene Therapy in Subjects With Stargardt Disease(STGD1)

NCTID	NCT06300476 (View at clinicaltrials.gov)
Description	The purpose of the study is to assess the safety and efficacy of JWK006 in Stargardt Disease(STGD1). JWK006 is packed by adeno-associated virus vector that expressing ABCA4 gene. (Show More)
Indication	Stargardt Disease (Juvenile Macular Degeneration)
Compound Name	JWK006
Sponsor	West China Hospital
Funder Type	Other
Status	Active not recruiting
Enrollment Count	9

Target Gene/Variant	ABCA4
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	AAV8
Editor Type
Dose 1	Undisclosed low dose
Dose 2	Undisclosed medium dose
Dose 3	Undisclosed high dose
Dose 4
Dose 5

No.of Trial Sites	1
Locations	China

Has US IND	False
Recent Updates	Investigator-initiated trial conducted at West China Hospital