FDA Approves Revolutionary CRISPR Therapy for Sickle Cell Anemia
The FDA has approved two gene therapies to treat sickle cell disease, including the first CRISPR/Cas9-based treatment in the US. Sickle cell disease is a debilitating condition that affects around 100,000 people in the US, primarily African Americans. The new gene therapies, Casgevy and Lyfgenia, have shown high effectiveness in preventing vaso-occlusive events and crises associated with the disease. Casgevy works by turning on the production of fetal hemoglobin, which prevents the deformation of red blood cells. Lyfgenia uses a Lentiviral vector to insert genes for a modified type of hemoglobin that is anti-sickling. Both therapies have shown promising results in clinical trials and are approved for patients aged 12 years and older.
- FDA approves first CRISPR therapy—here's how it works against sickle cell Ars Technica
- F.D.A. Approves 2 Sickle Cell Treatments, One Using CRISPR Gene Editing The New York Times
- FDA approves groundbreaking sickle cell anemia treatment NBC News
- A Sickle Cell Breakthrough Is Here. Now the Hard Part. Bloomberg
- First-ever gene editing therapies approved The Hill
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