To Evaluate the Clinical Study of GS1191-0445 Injection in the Treatment of Hemophilia a

NCTID	NCT06833983 (View at clinicaltrials.gov)
Description	This study is a single-arm, open-label, multicenter study evaluating the efficacy and safety of GS1191-0445 injection as a single dose in Chinese subjects with hemophilia A. GS1191-0445 is an AAV8-based gene therapy vector designed to express B-domain deleted human factor VIII (FVIII) under the regulation of a human liver-specific promoter. Following a single intravenous administration, AAV8 targets hepatocytes and facilitates the specific expression and secretion of FVIII into the bloodstream. (Show More)
Development Status	Active
Indication	Hemophilia A
Disease Ontology Term	DOID:12134
Compound Name	GS1191-0445
Sponsor	Gritgen Therapeutics Co., Ltd.
Funder Type	Industry
Recruitment Status	Not yet recruiting
Enrollment Count	50
Results Posted	Not Available

Target Gene/Variant	F8 (BDD variant)
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intravenous
Drug Product Type	Viral vector
Target Tissue/Cell	Hepatocyte
Delivery System	Viral transduction
Vector Type	AAV8
Editor Type
Dose 1	2E12 vg/kg
Dose 2	4E12 vg/kg
Dose 3
Dose 4
Dose 5

No.of Trial Sites	11
Locations	China

Has US IND	False
FDA Designations
Recent Updates	First patient dosed November 2023