βAS lentiviral vector

The βAS lentiviral vector is a gene therapy candidate designed for the treatment of sickle cell disease (SCD). It utilizes a lentiviral vector to deliver an anti-sickling β-globin transgene (βAS) into autologous hematopoietic stem/progenitor cells (HSPCs). The primary goal is to produce therapeutic hemoglobin tetramers that prevent the polymerization of sickle hemoglobin (HbS) and the subsequent formation of sickle-shaped red blood cells. Advanced iterations of this vector employ a bifunctional strategy, combining gene addition of the βAS transgene with gene silencing via artificial microRNAs (amiRs). These amiRs target either the fetal hemoglobin (HbF) repressor BCL11A (to induce endogenous anti-sickling γ-globin) or the sickle β-globin (HBB) mRNA (to directly reduce HbS levels), thereby enhancing the overall proportion of therapeutic hemoglobin and improving clinical outcomes.

Other names

anti-sickling beta-globin lentiviral vector