LentiHBBT87Q

LentiHBBT87Q is a gene therapy product that uses a lentiviral vector to deliver a modified human β-globin gene (βA-T87Q) into autologous hematopoietic stem cells (CD34+). The vector is designed to restore functional β-globin expression in patients with transfusion-dependent β-thalassaemia. The drug is administered by harvesting a patient’s hematopoietic stem cells, ex vivo transduction with LentiHBBT87Q, conditioning with busulfan, and reinfusion of the modified cells. The goal is to achieve transfusion independence by sustained expression of exogenous, functional adult hemoglobin (HbAT87Q), thereby correcting the underlying β-globin deficiency that causes thalassaemia. Clinical studies indicate significant rates of transfusion independence, durable hemoglobin production, and a favorable safety profile in pediatric and adolescent populations.

Brand names

HGI-001 InjectionHGI001 InjectionHGI 001 Injection

Other names

LentiHBBT87QLentiHBBT-87QLentiHBBT 87QHGI-001HGI001HGI 001