New modalities in play for sickle cell and β thalassemia: Data Byte

Gene engineering and silencing technologies are making headway for hemoglobinopathies, with over a dozen programs in clinical testing for β thalassemia and sickle cell disease. The class’ first marketed product, however, highlights the commercial challenges that lie ahead.

On Tuesday, bluebird bio (NASDAQ:BLUE), whose ex vivo gene therapy Zynteglo betibeglogene autotemcel is approved in the EU to treat transfusion-dependent β thalassemia (TDT), elected to withdraw the therapy from the German market after failing to reach a pricing agreement with the country’s health authorities. Marketing of the therapy has been suspended following clinical holds in February on a product that uses the same vector...