Sickle cell gene therapies offer new hope, but access remains a major challenge
- The FDA approved Casgevy and Lyfgenia in 2023 as the first two gene therapies for patients with severe forms of sickle cell disease.
- High costs, a complex treatment process, provider availability and access to specialized treatment centers have slowed use.
- By the end of 2025, uptake remained limited, with 64 patients worldwide receiving Casgevy infusions and 115 U.S. patients treated with Lyfgenia since its approval.