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The betibeglogene autotemcel (beti-cel) gene therapy caused paediatric and adult beta thalassaemia patients to achieve transfusion independence lasting over two years.
Bluebird bio has announced that its gene therapy Zynteglo (autologous CD34+ cells encoding βA-T87Q-globin gene) has been granted conditional marketing authorisation in Europe for certain patients 12 years and older with transfusion-dependent β-thalassaemi
US-based therapeutics developer bluebird bio has secured positive recommendation from a European Medicines Agency (EMA) panel for the approval of its first gene therapy Zynteglo to treat certain patients with transfusion-dependent β-thalassemia (TDT).
Bluebird Bio has been singing the praises of two new therapies at the annual American Society of Hematology, but although the data look solid so far, investors don’t seem to be fully on board with the program.......