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Man Cured Of Sickle Cell Disease In New York Thanks To New Gene Therapy
In a groundbreaking medical breakthrough, a 21-year-old man in New York has been cured of sickle cell disease following treatment with a pioneering gene therapy. Sebastien Beauzile received the Lyfgenia infusion on December 17th, 2024, and has not exhibited any symptoms of the debilitating condition since, leading his doctors to declare him “cured.”
Sickle cell anemia is a serious genetic disorder that primarily affects Black and Hispanic populations, causing numerous health complications including blood clots, strokes, and significantly reducing one’s lifespan by approximately 20 years on average. Historically, treatment options for the disease have been limited to symptom management, rather than a cure.
However, this innovative gene therapy, developed by Bluebird Bio, has now provided new hope for patients suffering from this devastating condition. Lyfgenia works by extracting a patient’s own blood stem cells, genetically modifying them using a virus to insert functional hemoglobin genes, and then administering chemotherapy to clear out the defective red blood cells before reinfusing the newly modified ones into the body. The resulting red blood cells are completely normal.
Sebastien Beauzile is not the first individual to benefit from this groundbreaking therapy in the United States. Lyfgenia and another genetically modified cell therapy, Casgevy (Vertex pharmaceuticals), were both FDA-approved in late 2023 for the treatment of sickle cell anemia in patients aged 12 and older, following impressive clinical trial results.
In a remarkable outcome, the Lyfgenia trial achieved a complete resolution of symptoms in 88% of 32 patients within 6-18 months after receiving the drug. Despite this stunning success, concerns regarding accessibility have emerged due to the therapy’s astronomical cost. Lyfgenia is priced at an eye-watering $3.1 million per treatment, while Casgevy costs $2.2 million.
