Welcoming the Era of Gene Editing in Medicine

This perspective piece reflects on the landmark approval of exagamglogene autotemcel (exa-cel) for sickle cell disease and transfusion-dependent β thalassemia, marking the beginning of clinical gene editing. The author traces the scientific journey from CRISPR-Cas9’s discovery to its therapeutic application, highlighting key milestones in globin gene regulation and the targeting of BCL11A to reactivate fetal hemoglobin. While celebrating the achievement, the article emphasizes challenges ahead accessibility, cost, and scalability and advocates for in vivo delivery platforms and global equity in genetic medicine.

This perspective piece reflects on the landmark approval of exagamglogene autotemcel (exa-cel) for sickle cell disease and transfusion-dependent β thalassemia, marking the beginning of clinical gene editing. The author traces the scientific journey from CRISPR-Cas9’s discovery to its therapeutic application, highlighting key milestones in globin gene regulation and the targeting of BCL11A to reactivate fetal hemoglobin. While celebrating the achievement, the article emphasizes challenges ahead accessibility, cost, and scalability and advocates for in vivo delivery platforms and global equity in genetic medicine.