Lillian Ren

Advances and Challenges in the Treatment of Sickle Cell Disease Using Gene Therapy

Started Apr. 22, 2026

Abstract or project description

Sickle cell disease (SCD) is a hereditary blood disorder that affects nearly 8 million people worldwide. It causes abnormal hemoglobin production, worsens quality of life through chronic anemia and vaso-occlusive events, and reduces life expectancy. Non-curative treatments have been established to help manage the symptoms of SCD, while curative therapies such as allogeneic hematopoietic stem cell transplantation face limitations from a lack of donor availability and complications from graft rejection. In recent years, early phase clinical trials have been conducted to analyze the efficacy and safety of using new gene editing based technologies as alternative cures for SCD. This paper will assess these advances with a focus on therapies including exagamglogene autotemcel and lovotibeglogene autotemcel, as well as variations of CRISPR-based HSPC therapy using Cas9 and Cas12a. Clinical studies have found that these therapies are able to consistently stabilize patient’s hemoglobin levels and effectively reduce vaso-occlusive events. Despite these successes, continued research is necessary to monitor the long-term safety of the different forms of gene therapy, address their high costs and limited accessibility, and further improve the safety of their pre-conditioning processes.