On December 8, 2023, the U.S. Food and Drug Administration (FDA) approved two new gene therapies for the treatment of sickle cell disease. [2] Casgevy™ is indicated for the treatment of sickle cell disease in patients 12 years and older with recurrent vaso occlusive crises (VOCs). [4]Sickle cell disease is an inherited blood disorder caused by a single mutated gene. These red blood cells then get caught in blood vessels, blocking the blood vessels. [5] Sickle cell disease can cause severe pain and organ damage, and can lead to life-threatening disabilities or death.

Source: New York Times December 20, 2023 22:02 UTC