Kanter J, Walters MC, Krishnamurti L, et al. - In sickle cell disease patients, a sustained production of HbA ^T87Q (an antisickling hemoglobin) in most red cells was seen after one-time treatment with LentiGlobin. This subsequently resulted in decreased hemolysis and complete resolution of severe vaso-occlusive events.

• Gene therapy with the use of LentiGlobin (bb1111; lovotibeglogene autotemcel) comprises autologous transplantation of hematopoietic stem and progenitor cells transduced with the BB305 lentiviral vector encoding a modified β-globin gene, which generates HbA ^T87Q .

• In this unprespecified interim analysis, safety and efficacy of LentiGlobin was assessed in 35 patients with sickle cell disease; they were given a LentiGlobin infusion and were observed for 17.3 months (range, 3.7 to 37.6).

• An increase in median total hemoglobin level, from 8.5 g per deciliter at baseline to 11 g or more per deciliter from 6 months through 36 months, was evident post-infusion.

• Decrease in hemolysis markers was observed, and HbA ^T87Q contributed at least 40% of total hemoglobin and was distributed across a mean (±SD) of 85±8% of red cells.

• Of 25 patients who could be assessed, resolution of severe vaso-occlusive events was achieved in all, relative to a median of 3.5 events per year (range, 2.0 to 13.5) in the 24 months prior to enrollment.

• A mild adverse event occurred in three patients, with resolution within 1 week after onset.

• No cases of hematologic malignancy occurred during up to 37.6 months of follow-up.