Gene Therapy Breakthrough: 21-Year-Old Cured of Sickle Cell Anemia in US

In a groundbreaking medical achievement, 21-year-old Sebastien Beauzile from Laurelton, has become the first person in New York State to be cured of sickle cell anemia using the innovative Lyfgenia gene therapy.

This pioneering treatment offers new hope for individuals battling the debilitating disease, Forbes reports on Sunday.

Beauzile’s journey with sickle cell anemia began at four months old, with frequent hospitalizations due to intense pain crises. 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, works by extracting the patient’s own blood stem cells, genetically modifying them, and then reintroducing these modified cells into the patient’s bloodstream.

Dr. Jeffrey Lipton, director of pediatric hematology oncology and stem cell transplantation at Cohen Children’s Medical Center, hailed the therapy as a cure, stating, “Other drugs modify the disease, but this is a cure… I suspect this will replace bone marrow transplants in time.”

Beauzile underwent the treatment process, which included chemotherapy, and received the genetically modified cells in late December 2024. By January 13, 2025, he reported feeling like a new person, saying, “When I got my cells, it feels like a second birthday.”

Since the therapy, Beauzile has resumed activities previously hindered by his condition and now aspires to pursue a medical career… aiming to support children facing similar health challenges. Expressing his gratitude, he remarked, “I’m not in pain anymore.”

The FDA’s approval of Lyfgenia in December 2023 marked a significant milestone in the treatment of sickle cell disease.

This gene therapy offers a one-time, single-dose infusion as part of a hematopoietic (blood) stem cell transplant, providing a potential cure for patients aged twelve years and older with a history of vaso-occlusive events.

Dr. Banu Aygun emphasized the transformative potential of Lyfgenia, stating, “When gene therapy became available… so that he can dramatically change his…”

While Lyfgenia is priced at $3.1 million per treatment, raising questions about accessibility, its success in treating Beauzile and other patients offers hope for a brighter future for those battling sickle cell anemia. As Dr. Lipton noted, “Sickle cell is a debilitating, often-overlooked disease. But treatments like Lyfgenia are changing that.”