bluebird bio Announces FDA Approval of LYFGENIA™ for Sickle Cell Disease
bluebird bio, Inc. has announced the U.S. Food and Drug Administration has approved LYFGENIA™ (lovotibeglogene autotemcel), also known as lovo-cel, as a one-time intravenous suspension gene therapy that has the potential to resolve vaso-occlusive events (VOC’s) and is custom-designed to treat the underlying cause of sickle cell disease (SCD).
Lyfgenia uses a replication-incompetent, self inactivating lentiviral vector (LVV) to add functional copies of the beta-globin gene to the patient’s own blood stem cells, which results in the production of anti-sickling hemoglobin that may decrease or stop vaso-occlusive events. It is made specifically for each patient. Patients are admitted to a treatment center during this process.
SCD is a genetic, inherited, lifelong disease caused by an alteration in one of the genes in the red blood cell, the beta-globin gene, that causes the normal disc shaped red cells to take the shape of a sickle, causing anemia and VOCs, like a pain crisis.
VOCs are when sickled red blood cells block blood flow, depriving tissues of oxygen. Lyfgenia uses a common, well-studied viral vector to deliver genetic modifications to a patient’s blood stem cells, so no donor is needed.
