CHMP backs approval of bluebird's beta-thalassemia gene therapy

EMA's CHMP recommended conditional approval of bluebird's gene therapy Zynteglo to treat transfusion-dependent β-thalassemia. The therapy is not yet approved in any territory.

The committee backed Zynteglo's approval in patients ages 12 and older who do not have a β-0/β-0 genotype, and for whom hematopoietic stem cell transplantation (HSCT) is appropriate but a matched donor is unavailable. The LentiGlobin-based therapy consists of autologous CD34⁺ stem cells transduced ex vivo with a lentiviral vector delivering the human β globin gene...