FDA Approves First CRISPR-Based Therapy: Cevi

Overview

• Cevi: New CRISPR-based therapy approved by FDA
• Developed by Vertex Pharmaceuticals and CRISPR Therapeutics
• Milestone in genetic medicine
• Treats sickle cell disease and beta-thalassemia
• Affects hundreds of thousands, mainly of African and Asian descent

How It Works

• Both disorders arise from hemoglobin mutations
• Sickle cell disease: Mutated hemoglobin deforms red blood cells into a sickle shape, causing blockages
• Beta-thalassemia: Insufficient hemoglobin production, leading to low blood oxygen levels
• Cevi's Function: Edits gene in patient’s blood stem cells to resume fetal hemoglobin production
  • Fetal hemoglobin: Produced during fetal development, carries oxygen efficiently

Treatment Process

1. Blood Collection: Patient’s blood cells collected
2. Isolation: Stem cells isolated
3. Preparation: Cells prepped for gene editing
4. CRISPR Components: Added to cells
   • Cutting enzyme: C9
   • Guide RNA: Directs editing molecule
5. Electroporation: Uses electricity to introduce materials into cells
6. Growth and Harvesting: Edited cells grown and harvested
7. Freezing: Cells frozen until infusion
8. Infusion: Therapy infused back into patient

Challenges

• Chemotherapy Required: Remove existing bone marrow cells
  • Side effects: Hair loss, mouth sores, infertility
• Hospital Stay: Weeks required for recovery
• Time and Cost: Process takes months and costs millions of dollars