Gene Therapy Offers New Sickle Cell Cure in the US
Sebastien Beauzile, a 21-year-old from Laurelton, Long Island, has become the first person in New York State to be cured of sickle cell anemia using the groundbreaking Lyfgenia gene therapy.
Administered at Cohen Children’s Medical Center, this innovative treatment provides hope for many suffering from the painful genetic disorder, Forbes reported on Sunday.
A Lifelong Battle
Sickle cell anemia causes abnormally shaped red blood cells, leading to severe pain and potential organ damage. Beauzile, diagnosed at just four months old, endured frequent hospitalizations due to intense pain crises, which he described as “10 out of 10” in severity.
Gene Therapy Breakthrough
Developed by Bluebird Bio and approved by the FDA in December 2023, Lyfgenia works by extracting a patient’s blood stem cells, genetically modifying them to include functional hemoglobin genes, and reinfusing them into the bloodstream. This cutting-edge approach marks a major step forward in treating sickle cell disease.
