FDA Considers Approval of CRISPR-Based Treatment to Combat Sickle Cell Disease

4 months ago
The FDA's independent advisory committee is evaluating a gene therapy treatment called exa-cel that uses CRISPR gene editing to potentially cure sickle cell disease. Sickle cell disease is a genetic blood disorder with no cure where red blood cells become misshapen and can cause organ damage, severe pain episodes, and early death. Exa-cel therapy alters a patient's own stem cells using CRISPR to fix the genetic mutation that causes sickle cell disease. The FDA will make a final approval decision on exa-cel therapy by December 8th after considering the committee's perspectives.