New York man cured of sickle cell disease

Sebastien Beauzile, a 21-year-old from New York, has been cured of sickle cell anemia after receiving a new gene therapy called Lyfgenia. His treatment took place on December 17, 2024, and he has shown no symptoms since. Lyfgenia, developed by Bluebird Bio, modifies the patient's blood stem cells to produce normal red blood cells. This therapy is a significant advancement compared to previous treatments, which only managed symptoms or required risky bone marrow transplants. Both Lyfgenia and another therapy, Casgevy, were FDA-approved in late 2023. However, the high cost of Lyfgenia at $3.1 million raises concerns about accessibility for patients with sickle cell disease.