Safety and Efficacy Trial of HG004 for Leber Congenital Amaurosis Related to Rpe65 Gene Mutations (STAR)

NCTID	NCT05906953 (View at clinicaltrials.gov)
Description	The purpose of the study is to determine whether HG004 as gene therapy is safe and effective for the treatment of Leber Congenital Amaurosis caused by mutations in RPE65 gene. (Show More)
Indication	Inherited Retinal Disease Associated With RPE65 Mutations
Compound Name	HG004
Sponsor	HuidaGene Therapeutics Co., Ltd.
Funder Type	Industry
Status	Recruiting
Enrollment Count	20

Target Gene/Variant	RPE65
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	AAV9
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	3
Locations	United States,China

Has US IND	True
Recent Updates	FDA granted RPDD on 8/7/23