Following long-term efficacy and safety data from the beti-cel trials, how do you approach gene therapy for eligible patients with transfusion-dependent β-thalassemia?  

The results with beti-cel are excellent. So are the results with exa-cel. These two gene therapies use different approaches to modifying hemoglobin production in erythroid stem cells. Beti-cel adds a modified hemoglobin that resembles fetal hemoglobin using viral-mediated transduction, whereas exa-c...

The long-term data for gene therapy (both beti-cel and exa-cel) in transfusion-dependent thalassemia have been very reassuring. The clinical trial participants have all achieved transfusion independence (if you only include the beti-cel subjects who received the optimized manufacturing). There have ...