Representational image of blood capillaries in sickle-cell disease (left)|Scientific Animations|CC BY-SA 4.0
The Food and Drug Administration (FDA) has approved Casgevy, the first gene-editing therapy ever to be used in humans for treating sickle-cell disease.
It uses the Nobel-prize-winning CRISPR gene-editing tool. The treatment collects and then genetically modifies a patient’s bone marrow stem cells that are later infused into the patient.
Prevalent among Black people, sickle cell disease is a genetic disorder in which red blood cells contort into a sickle-shaped cell disease that affects hemoglobin. It causes pain, organ damage, and early death. The disease affects around 100,000 in the US and over 20 million globally.
But
The therapy takes months to complete, compromises the immune system, and costs $2.2 million per patient.
Additionally
The FDA also approved Bluebird Bio’s Lyfgenia, the second treatment for sickle-cell disease. But it comes with a black-box warning that it can cause rare blood cancer. The therapy costs $3.1 million.