FDA clears first CRISPR treatment for a second disease, beta thalassemia

The FDA has approved Casgevy, a CRISPR-based medicine, for treating beta thalassemia in individuals over 12. Originally approved for sickle cell disease, this innovative treatment uses CRISPR/Cas9 to modify stem cells, enhancing hemoglobin production and alleviating symptoms of blood disorders.

Beta thalassemia, a genetic condition causing inadequate hemoglobin production, leads to anemia and reduced life expectancy. Traditional management involves frequent blood transfusions, which can cause complications like iron overload. Casgevy offers a new treatment option, potentially reducing the need for transfusions.

Despite its effectiveness, Casgevy is priced at $2.2 million, posing affordability challenges. However, its approval marks a significant advancement in treating genetic disorders, highlighting the promise of CRISPR technology in medical science.