Sickle cell disease fi nally getting its due

Biologies license applications were filed recently for two potentially curative gene therapies in sickle cell disease (SCD), representing an astonishing advance for a condition characterized by crippling pain and a shortened life span even with current treatments. Cystic fibrosis has received much more funding than sickle cell disease, and providers have reported in surveys that they do not feel comfortable with managing care for this disease. In July 2023, researchers at the University of Pennsylvania and Children's Hospital of Philadelphia reported positive findings in the journal Science for a messenger RNA approach to gene therapy that does not require myeloablation.