The First CRISPR-Based Medicine Expected to Win FDA Approval

A groundbreaking week in genome editing is expected as the first CRISPR-based medicine, Cass Jeavy, developed by CRISPR Therapeutics and Vertex Pharmaceuticals, approaches FDA approval. Cass Jeavy aims to treat sickle cell disease by using CRISPR-Cas9 to increase the production of fetal hemoglobin, which helps prevent sickled red blood cells. The selection of sickle cell disease as the target for this groundbreaking development can be attributed to the suitability of CRISPR's capabilities for addressing this particular condition. This achievement marks a significant milestone in the field of genome editing and paves the way for future CRISPR-based therapies.