This is a non randomized, open label, multi site, single dose, Phase 1/2 study in up to 15 adults with β thalassemia major who receive at least 100 mL/kg/year of packed red blood cells (pRBCs) or ≥8 transfusions of pRBCs per year. The study will evaluate the safety and efficacy of autologous hematopoietic stem cell transplantation (HSCT) using LentiGlobin® BB305 Drug Product (autologous CD34+ hematopoietic stem cells transduced with LentiGlobin® BB305 lentiviral vector encoding the human βA T87Q globin gene and stored in cryopreservative solution in the final immediate container for the intended medical use).

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More information: clinicaltrials.gov