Sickle cell gene therapies offer new hope, but access remains a major challenge

For decades, people living with sickle cell disease have endured a lifetime of hospital visits, medications and blood transfusions to manage a painful genetic blood disorder that can shorten life expectancy by up to 20 years . But a long-awaited breakthrough came in 2023, when the U.S. Food and Drug Administration approved Casgevy and Lyfgenia, the first two gene therapies for patients with severe forms of the disease. Patient advocates and clinicians say the recent advancements in treatment could mark the beginning of a new era in sickle cell care, one that may free thousands from recurrent episodes of debilitating pain and improve long-term health outcomes.