Article
Betibeglogene autotemcel gene therapy in patients with transfusion-dependent, severe genotype β-thalassaemia (HGB-212): a non-randomised, multicentre, single-arm, open-label, single-dose, phase 3 trial.
Lancet (London, England) - 30 Nov 2024
Kwiatkowski Janet L, Walters Mark C, Hongeng Suradej, Yannaki Evangelia, Kulozik Andreas E, Kunz Joachim B, Sauer Martin G, Thrasher Adrian J, Thuret Isabelle, Lal Ashutosh, Tao Ge, Ali Shamshad, Thakar Himal L, Elliot Heidi, Lodaya Ankit, Lee Ji, Colvin Richard A, Locatelli Franco, Thompson Alexis A
Abstract excerpt
BACKGROUND: Transfusion-dependent β-thalassaemia (TDT) is a severe disease, resulting in lifelong blood transfusions, iron overload, and associated complications. Betibeglogene autotemcel (beti-cel) gene therapy uses autologous haematopoietic stem and progenitor cells (HSPCs) transduced with BB305 lentiviral vector to enable transfusion independence. METHODS: HGB-212 was a non-randomised, multicentre, single-arm,...
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