21-year-old Sebastien Beauzile from Laurelton, Long Island, has become the first person in New York State to be cured of sickle cell anaemia using the innovative Lyfgenia gene therapy.
This pioneering treatment, administered at Cohen Children’s Medical Center, offers a beacon of hope for countless individuals battling this debilitating disease, Forbes reports on Sunday.
Sickle cell anemia is a genetic disorder characterized by misshapen red blood cells that can obstruct blood flow, leading to severe pain and potential organ damage.
Beauzile’s battle with this condition began at just four months old, with frequent hospitalizations due to intense pain crises.
According to New York Post, he described the agony as “10 out of 10,” with his back feeling “like somebody was either pulling on it or hanging on it,” and his chest feeling “like somebody is sitting on it.”
Lyfgenia, developed by Bluebird Bio, represents a significant advancement in gene therapy.
Approved by the U.S. Food and Drug Administration (FDA) in December 2023, Lyfgenia works by extracting the patient’s own blood stem cells, genetically modifying them using a virus to insert functional copies of the hemoglobin gene, and then reintroducing these modified cells into the patient’s bloodstream