First launch of bluebird bio's Zynteglo

13 January 2020

US gene therapy company bluebird bio (Nasdaq: BLUE) has announced the first launch anywhere of Zynteglo (autologous CD34+ cells encoding βA-T87Q-globin gene).

The one-time gene therapy has been launched in Germany for patients 12 years and older with transfusion-dependent β-thalassaemia (TDT) who do not have a β0/β0 genotype, for whom hematopoietic stem cell (HSC) transplantation is appropriate but a human leukocyte antigen (HLA)-matched related HSC donor is not available.

TDT is a severe genetic disease caused by mutations in the β-globin gene that result in reduced or significantly reduced hemoglobin (Hb).

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