FDA Approves Groundbreaking Gene Therapies for Sickle Cell Disease, Revolutionizing Treatment Options
The FDA has approved two groundbreaking gene therapies, Casgevy and Lyfgenia, for the treatment of sickle cell disease (SCD) in patients aged 12 and older. Casgevy is the first FDA-approved therapy to use CRISPR/Cas9 genome editing technology, while Lyfgenia utilizes a lentiviral vector for genetic modification. Both therapies involve modifying the patient's own blood stem cells and delivering them back as a one-time infusion. These treatments aim to increase the production of fetal hemoglobin or introduce a gene-therapy derived hemoglobin to prevent the sickling of red blood cells. The approvals mark a significant advancement in gene therapy and offer hope for individuals with SCD, a rare and debilitating blood disorder.
- FDA Approves First Gene Therapies to Treat Patients with Sickle Cell Disease FDA.gov
- Gene editing could be a game changer for patients with sickle cell disease CBS Mornings
- F.D.A. Approves 2 Sickle Cell Treatments, One Using CRISPR Gene Editing The New York Times
- A Sickle Cell Breakthrough Is Here. Now the Hard Part. Bloomberg
- The Download: inside the first CRISPR treatment, and smarter robots MIT Technology Review
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