LYFGENIA is a one-time gene therapy to treat sickle cell disease in
patients 12 years of age or older and a history of vaso-occlusive events. Sickle cell
disease 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 vaso-occlusive events, like a
pain crisis. LYFGENIA is made specifically for each patient, using the patient’s own
blood stem cells (from which red blood cells are produced). It adds functional copies of
the beta-globin gene to your cells leading to production of anti-sickling hemoglobin that
may decrease or stop vaso-occlusive events.