Zynteglo
Zynteglo is a gene therapy intended for people with beta thalassemia, a rare inherited condition that results in lower production of hemoglobin and red blood cells, which help transport oxygen around the body. The treatment modifies blood cells so that they can produce functional beta-globin, a component of hemoglobin.
Approved by the FDA in August 2022, the single-dose treatment is expected to cost $2.8 million per patient—seemingly the highest price yet for an individual course of medication. If it works as intended, though, it would replace the lifelong blood transfusions and extended hospital stays that patients with beta thalassemia typically require.
