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Bluebird bio’s beta-thalassaemia gene therapy rejected by NICE

February 12, 2021

Beti-cel, marketed as Zynteglo in Europe, is a gene therapy intended for the treatment of transfusion-dependent beta-thalassaemia (TDT) in people aged 12 years and older who do not have a beta0/beta0 genotype.

It is indicated for TDT patients when haematopoietic stem cell transplantation (HSCT) is appropriate but there is no suitable donor.

TDT is the most severe form of thalassaemia, a condition wherein an inherited faulty gene leads to the inability of the body to produce normally functioning haemoglobin.

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