Extending Gene Medicines to All in Need

This editorial discusses the transformative potential of gene therapies like exagamglogene autotemcel (exa-cel) and lovotibeglogene autotemcel (lovo-cel) for sickle cell disease and transfusion dependent β-thalassemia. While these therapies mark major scientific progress, their accessibility remains limited due to high costs, complex procedures, and concentration in high-income countries. The authors advocate for in vivo delivery platforms, global regulatory harmonization, and collaborative efforts to develop affordable, scalable solutions. They highlight initiatives by the Gates Foundation and NIH to drive equitable access to gene-based cures worldwide.

This editorial discusses the transformative potential of gene therapies like exagamglogene autotemcel (exa-cel) and lovotibeglogene autotemcel (lovo-cel) for sickle cell disease and transfusion dependent β-thalassemia. While these therapies mark major scientific progress, their accessibility remains limited due to high costs, complex procedures, and concentration in high-income countries. The authors advocate for in vivo delivery platforms, global regulatory harmonization, and collaborative efforts to develop affordable, scalable solutions. They highlight initiatives by the Gates Foundation and NIH to drive equitable access to gene-based cures worldwide.