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.