Betibeglogene autotemcel gene therapy for non–β⁰/β⁰ genotype β-thalassemia

• Main Authors: Franco Locatelli, Alexis A. Thompson, Janet L. Kwiatkowski, John B. Porter, Adrian J. Thrasher, Suradej Hongeng, Martin G. Sauer, Isabelle Thuret, Ashutosh Lal, Mattia Algeri, Jennifer Schneiderman, Timothy S. Olson, Ben Carpenter, Persis J. Amrolia, Usanarat Anurathapan, Axel Schambach, Christian Chabannon, Manfred Schmidt, Ivan Labik, Heidi Elliot, Ruiting Guo, Mohammed Asmal, Richard A. Colvin, Mark C. Walters
• Other Authors: Ramathibodi Hospital
• Format: Article
• Published: 2022
• Subjects: Medicine
• Online Access: https://repository.li.mahidol.ac.th/handle/123456789/74805

BACKGROUND Betibeglogene autotemcel (beti-cel) gene therapy for transfusion-dependent β-thalassemia contains autologous CD34+ hematopoietic stem cells and progenitor cells transduced with the BB305 lentiviral vector encoding the β-globin (βA-T87Q) gene. METHODS In this open-label, phase 3 study, we...