Gene Therapy for Haemophilia A.

NCTID	NCT03001830 (View at clinicaltrials.gov)
Description	The GO-8 study focuses on assessing safety and efficacy of gene therapy for patients with severe haemophilia A (Show More)
Indication	Hemophilia A
Compound Name	AAV2/8-HLP-FVIII-V3
Sponsor	University College, London
Funder Type	Other
Status	Recruiting
Enrollment Count	18

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	AAV2/8
Editor Type
Dose 1	6 x 10^11 vg/kg
Dose 2	2 x 10^12 vg/kg
Dose 3	4 x 10^12 vg/kg
Dose 4	6 x 10^12 vg/kg
Dose 5

No.of Trial Sites	4
Locations	United States,United Kingdom

Has US IND	True
Recent Updates