Gene Therapy for Chinese Hemophilia A

NCTID	NCT04728841 (View at clinicaltrials.gov)
Description	IHBDH-GTHA-2020 is an open- label, non- randomized study to evaluate the safety, tolerability and kinetics of a single intravenous infusion of GS001 in hemophilia A subjects with \<1 IU/dl residual FVIII levels. (Show More)
Development Status	Active
Indication	Hemophilia A
Disease Ontology Term	DOID:12134
Compound Name	GS001
Compound Description	AAV8-BBD F8
Sponsor	Institute of Hematology & Blood Diseases Hospital, China
Funder Type	Other
Recruitment Status	Recruiting
Enrollment Count	12
Results Posted	Not Available

Target Gene/Variant	F8
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
Delivery System	Viral transduction
Vector Type	AAV8
Editor Type
Dose 1	2E12 vg/kg
Dose 2	4E12 vg/kg
Dose 3	Up to 2E13 vg/kg
Dose 4
Dose 5

No.of Trial Sites	1
Locations	China