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CHMP recommendation for bluebird bio's gene therapy in β-thalassaemia

Massachusetts-based bluebird bio has taken a significant leap forward in its pursuit of regulatory authorisation for its first gene therapy, Zynteglo (autologous CD34+ cells encoding β-globin AT87Q gene), securing recommendation from the EMA’s Committee for Medicinal Products for Human Use (CHMP) ) in the treatment of transfusion-dependent β-thalassaemia (TDT).

Specifically, the recommendation referred to the treatment of the blood disorder in patients over 12 years of age who do not have a β0/β0 genotype, for whom haematopoietic stem cell (HSC

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