After collection of mobilised peripheral blood samples, the patient's autologous cells,enriched for CD34+ HSCs, undergo ex vivo transduction with lentiviral vector encoding βA-T87Q-globin to BD211 finished product,which is then infused intravenously into the patient after myeloablative busulfan conditioning to prepare bone marrow niches for engraftment of the HSCs.~After discharge, subjects will be followed monthly, at a minimum, for 6 months and thereafter every 3 months for the remainder of the 24 months post-transplant.~Evaluation will include Routine and special biological testing at regular intervals, collection of AEs and concomitant medications, and evaluation of disease specific biological and clinical parameters.~Subjects will then be enrolled in a long-term follow-up protocol with annual evaluations for an additional 13 years post-transplant.~The long-term follow-up study will focus on long-term safety, with an emphasis on integration site analysis, and long-term efficacy.~This study will end when the last subject completes the Month 24 visit or discontinues from the study.
This is a Phase 1,open label,safety,and efficacy study in subjects with non-β0/β0 TDT β-thalassemia Major by transplanting BD211 drug product which is for autologous use only,via a single IV administration.