Lovo‐cel gene therapy for sickle cell disease: Treatment process evolution and outcomes in the initial groups of the HGB ‐206 study

Abstract excerpt

Abstract lovo‐cel (bb1111; LentiGlobin for sickle cell disease [SCD]) gene therapy (GT) comprises autologous transplantation of hematopoietic stem and progenitor cells transduced with the BB305 lentiviral vector encoding a modified β‐globin gene (βA‐T87Q) to produce anti‐sickling hemoglobin (HbAT87Q). The efficacy and safety of lovo‐cel for SCD are being evaluated in the ongoing phase 1/2 HGB‐206 study (...

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