FDA Approves First Gene Therapies to Treat Patients with Sickle Cell Disease

 

• The FDA approved two groundbreaking cell-based gene therapies, Casgevy and Lyfgenia, for treating sickle cell disease (SCD) in patients aged 12 and older.
• SCD affects around 100,000 people in the U.S., primarily African American and Hispanic American populations, causing severe pain and organ damage due to mutated hemoglobin.
• Casgevy is the first FDA-approved therapy using CRISPR/Cas9 technology, modifying patients’ stem cells to increase fetal hemoglobin production and prevent sickling of red blood cells.
• Lyfgenia, another approved therapy, modifies stem cells to produce a special hemoglobin variant that reduces the risk of blood flow blockages.
• Both therapies use patients’ own modified stem cells in a one-time infusion after high-dose chemotherapy, with ongoing studies to monitor their safety and effectiveness.
• These approvals signify a significant step in innovative gene therapies targeting debilitating diseases like SCD, reflecting the FDA’s commitment to advancing safe and effective treatments.

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