Hematopoietic Stem Cell Transplantation Gene Therapy for Treatment of Severe Hemophilia A

NCTID	NCT04418414 (View at clinicaltrials.gov)
Description	This is a first-in-human, non-randomized, open label, single treatment, Phase 1 study in approximately 7 patients with severe hemophilia A. The study will evaluate gene therapy by transplantation of autologous CD34+ hematopoietic stem cells transduced ex vivo with the CD68-ET3 lentiviral vector. (Show More)
Indication	Hemophilia A
Compound Name	CD68-ET3
Sponsor	Expression Therapeutics, LLC
Funder Type	Industry
Status	Not yet recruiting
Enrollment Count	7

Target Gene/Variant	F8
Therapy Type	Gene transfer
Therapy Route	Ex-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intravenous
Drug Product Type	Autologous cells
Target Tissue/Cell	CD34+ cells
Delivery System	Viral transduction
Vector Type	LV
Editor Type
Dose 1	Transduced CD34+ cells
Dose 2
Dose 3
Dose 4
Dose 5

No.of Trial Sites
Locations

Has US IND	True
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