Gene Therapy for Transfusion Dependent Beta-thalassemia

NCTID	NCT02453477 (View at clinicaltrials.gov)
Description	This is a phase I/II study evaluating safety and efficacy of autologous hematopoietic stem cells genetically modified with GLOBE lentiviral vector encoding for the human beta-globin gene for the treatment of patients affected by transfusion dependent beta-thalassemia (Show More)
Development Status	Inactive
Indication	Beta-Thalassemia Major
Disease Ontology Term	DOID:0080771
Compound Name	OTL-300
Sponsor	IRCCS San Raffaele
Funder Type	Other
Recruitment Status	Unknown
Enrollment Count	10
Results Posted	Not Available

Target Gene/Variant	HBB
Therapy Type	Gene transfer
Therapy Route	Ex-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intraosseous
Drug Product Type	Autologous cells
Target Tissue/Cell	CD34+ cells
Delivery System	Viral transduction
Vector Type	LV
Editor Type	none
Dose 1	Target dose: 5E6 CD34+ cells/kg
Dose 2	Minimum dose: 2E6 CD34+ cells/kg
Dose 3	Maximum dose: 20E6 CD34+ cells/kg
Dose 4
Dose 5

No.of Trial Sites	1
Locations	Italy

Has US IND
FDA Designations
Recent Updates	Program discontinued by Orchard