LentiGlobin HPV569

LentiGlobin HPV569 is a first-generation ex vivo lentiviral gene therapy developed by bluebird bio for the treatment of beta-thalassemia and sickle cell disease. The therapy involves the transduction of autologous hematopoietic stem cells (HSCs) with the HPV569 lentiviral vector, which delivers a functional human beta-globin gene (HBB) variant containing the T87Q mutation (HbAT87Q). This modified hemoglobin is designed to restore functional hemoglobin levels in thalassemia patients and inhibit the polymerization of sickle hemoglobin (HbS) in patients with sickle cell disease. HPV569 served as the proof-of-concept predecessor to the more potent next-generation BB305 vector (used in Zynteglo and Lyfgenia). While active clinical development of the HPV569-based product has concluded, subjects from early trials (such as LG001) are being monitored in long-term follow-up studies to assess the durability and safety of the therapy.

Other names

HPV569 lentiviral vectorHPV-569 lentiviral vectorHPV 569 lentiviral vector