Base Editing of HBG1 and HBG2 Promoters for Sickle Cell Disease

Key Points

• To evaluate the efficacy of risto-cel in enhancing hemoglobin levels and reducing sickle hemoglobin in sickle cell disease.
• Administered risto-cel treatment to evaluate its effects on hemoglobin expression.
• Monitored engagement of patients to assess engraftment and expression of hemoglobin types.
• Assessed endpoints such as HbF and HbS levels post-treatment.
• Rapid engraftment of the treatment was observed.
• Durable expression of fetal hemoglobin (HbF) was confirmed.
• Significant reduction in sickle hemoglobin (HbS) levels associated with treatment.