The Food and Drug Administration (FDA) has approved CASGEVY™ (exagamglogene autotemcel) and LYFGENIA™ (lovotibeglogene autotemcel), the first two gene therapies for the treatment of sickle cell disease in patients 12 years and older with recurrent vaso-occlusive crises.
Researchers developed a proof-of-concept model for delivering gene editing tools to treat blood disorders, allowing for the modification of diseased blood cells directly within the body.
In this Q&A, hematologist Dr. Kwiatkowski discusses the Food and Drug Administration approval of beti-cel (Zynteglo®), a gene therapy for beta thalassemia.