Sickle cell disease can shorten life expectancy by up to 20 years, and in 2023 the FDA approved Casgevy and Lyfgenia, the first two gene therapies for severe forms of the disease. Patient advocates and clinicians say these advances could mark a new era of care that frees many from recurring painful episodes. However, access remains uneven, as costs, complex treatment processes, limited provider availability and differing access to specialized centers can keep eligible patients from treatment. The CDC estimates about 100,000 people in the U.S. have the disease, more than 90% of them non-Hispanic Black or African American. Gene therapy has reported high efficacy, with 88% to 94% of patients, while hydroxyurea, approved in 1998, halved the frequency of pain crises in studies.
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