Autologous CD34+ cells transduced with the BB305 lentiviral vector.
An initial phase 1/2 study [Northstar (HGB-204)] in 22 β-thalassaemia patients showed transfusion-independency for non-β0/β0 genotypes and 60% reduction in transfuions in β0/β0 genotype. The results were published in N Engl J Med, in April 19, 2018.
An ongoing phase 2 study (HGB-205) evaluates LentiGlobin gene therapy in TDT and severe SCD.
An ongoing phase 3 trial [Northstar-2 (HGB-207)] evaluates LentiGlobin gene therapy in TDT with non-β0/β0 genotypes.