EMA to review bluebird's thalassemia gene therapy

bluebird bio Inc. (NASDAQ:BLUE) said EMA accepted for review an MAA for LentiGlobin BB305 to treat patients with transfusion-dependent β-thalassemia and non-β-0/β-0 genotypes.

In August, EMA's CHMP granted accelerated access to the gene therapy, which comprises autologous CD34+ stem cells transduced ex vivo with a lentiviral vector delivering the human β globin gene. The candidate also has Priority Medicines (PRIME) and Orphan Drug designations in the EU, and breakthrough therapy and Orphan Drug designations in the U.S...