bluebird bio remains in a tight race with Vertex Pharmaceuticals Incorporated to bring the first gene-altering therapy to market for sickle cell disease, announcing on 24 April that it has filed a biologics license application (BLA) with the US Food and Drug Administration for the gene therapy lovotibeglogene autotemcel (lovo-cel) in patients with sickle cell disease ages 12 and older who have a history of vaso-occlusive events (VOEs).
The announcement from bluebird is good news for investors, some of whom have been concerned about a longer delay in filing after the company revealed on 29 March that it would not meet a goal to file lovo-cel by the end of the first quarter