lentiviral vector encoding T87Q beta-globin

The lentiviral vector encoding T87Q beta-globin is a gene therapy platform developed by bluebird bio for the treatment of beta-hemoglobinopathies, including beta-thalassemia and sickle cell disease (SCD). The platform utilizes a self-inactivating (SIN) lentiviral vector (primarily BB305) to deliver a modified human beta-globin gene (HBB) into a patient's own CD34+ hematopoietic stem cells ex vivo. The modified gene features a threonine-to-glutamine substitution at position 87 (T87Q), which provides anti-sickling properties similar to those of fetal hemoglobin (HbF) and prevents the polymerization of sickle hemoglobin (HbS). This technology is the basis for two FDA-approved autologous cell therapies: betibeglogene autotemcel (Zynteglo) for transfusion-dependent beta-thalassemia and lovotibeglogene autotemcel (Lyfgenia) for sickle cell disease. After the transduced cells are infused back into the patient, they engraft in the bone marrow and produce red blood cells that express functional T87Q-globin, thereby correcting the underlying genetic defect and reducing disease complications.

Brand names

ZyntegloLyfgenia

Other names

LentiGlobinbetibeglogene autotemcellovotibeglogene autotemcelβA-T87Q-globin lentiviral vector