Abstract

This is a single-arm, multi-site, single-dose, Phase 3 study in approximately 23 participants less than or equal to (<=) 50 years of age with transfusion-dependent β-thalassemia (tdt), also known as β-thalassemia major, who do not have a β0 mutation at both alleles of the hemoglobin β (hbb) gene. the study will evaluate the efficacy and safety of autologous hematopoietic stem cell transplantation (hsct) using lentiglobin bb305 drug product.>

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