FDA Approves Not One, But Two Sickle Cell Disease Gene Therapies

The FDA approved gene therapies from Vertex Pharmaceuticals and Bluebird Bio for sickle cell disease. Both treatments, Casgevy and Lyfgenia, use gene therapy to potentially cure the inherited blood disorder and target those aged 12 and older. Sickle Cell Disease is a rare, debilitating, and life-threatening blood disorder with significant unmet need. Healthy red blood cells are round and flexible, while those with sickle cell disease have rigid, crescent-shaped cells that block blood flow and cause painful episodes. Both therapies harvest stem cells, modify them in the lab, and present results of reduced vaso-occlusive crises. The treatments range in price and pose commercialization challenges.

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