Stem Cell Gene Therapy for Sickle Cell Disease

NCTID	NCT02247843 (View at clinicaltrials.gov)
Description	This Phase I clinical trial will assess the safety and initial evidence for efficacy of an autologous transplant of lentiviral vector modified peripheral blood for adults with severe sickle cell disease. (Show More)
Indication	Sickle Cell Disease
Compound Name	Lenti/G-βAS3-FB (contains anti-sickling mutations: T87Q, G16D, E22A)
Sponsor	Donald B. Kohn, M.D.
Funder Type	Other
Status	Active not recruiting
Enrollment Count	4

Target Gene/Variant	HBB
Therapy Type	Gene transfer
Therapy Route	Ex-vivo
Mechanism of Action	Overexpression of protective allele/gene
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	1
Locations	United States

Has US IND	True
Recent Updates	Uses the same βAS3-globin as NCT03964792