Gene-Modified Stem Cell Therapy for Subjects With Transfusion-dependent Beta-thalassemia

This is a prospective, dual-centre, single dose, Phase IIb, single arm, open label study. The proposed clinical trial involves a single infusion of autologous HSPCs genetically modified with the GLOBE lentiviral vector, using an improved transduction protocol in 9 patients affected by transfusion dependent Beta-Thalassemia. Four study phases are foreseen: 1. Screening phase, during which the conditions required by the clinical protocol for patients' inclusion/exclusion will be assessed after the signature of the informed consents/assents. Patients will be recruited from the two participating sites: IRCCS Ospedale San Raffaele (OSR), Department of Pediatric Immunohematology and adult Hematology (OSR Stem Cells Programme) (Milan) and IRCCS Ospedale Pediatrico Bambino Gesù (OPBG), Department of Haematology, Oncology and Gene and Cell Therapy (Rome). 2. Baseline phase, carried from the end of the screening phase to the day before the start of the conditioning regimen. 3. Treatment phase, from the first day of conditioning regimen until DP administration. 4. Follow-up phase: from DP administration until 2 years follow-up.