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)
Development Status	Active
Indication	Sickle Cell Disease
Disease Ontology Term	DOID:0081445
Compound Name	Lenti
Compound Alias	G-βAS3-FB
Compound Description	contains anti-sickling mutations: T87Q, G16D, E22A
Sponsor	Donald B. Kohn, M.D.
Funder Type	Other
Recruitment Status	Active not recruiting
Enrollment Count	4
Results Posted	Not Available

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	none
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
FDA Designations
Recent Updates	Uses the same βAS3-globin as NCT03964792