Exagamglogene Autotemcel for Severe Sickle Cell Disease
May 1, 2024
Very exciting development in CRISPR-based therapy for transfusion-dependent sickle cell disease (SSD). 100% of patients avoided hospitalization for occlusive crisis. Long-term risks and concern for off-target gene editing will remain a concern. An argument can be made that for frequently admitted patients that this could be cost effective.
