HomeHealthPanel Assures F.D.A. of Sickle Cell Cure's Safety for Patients Using CRISPR

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Panel Assures F.D.A. of Sickle Cell Cure’s Safety for Patients Using CRISPR

A group of experts announced on Tuesday that a revolutionary treatment for sickle cell disease is safe for clinical use, possibly leading to approval from the Food and Drug Administration (FDA) by December 8th. The treatment, known as exa-cel, is a joint development between Vertex Pharmaceuticals and CRISPR Therapeutics. The FDA had previously found the treatment to be effective, and the expert panel’s conclusion on its safety sends it to the FDA for approval. If approved, exa-cel would be the first medicine to treat a genetic disease using CRISPR gene-editing technology. The approval of exa-cel could open the doors to a series of new treatment options for sickle cell disease, including a gene therapy developed by Bluebird Bio. Sickle cell disease affects millions of people worldwide, with the majority being of African ancestry. The disease causes misshapen blood cells that can lead to strokes, organ damage, and excruciating pain. The new therapies are expected to be expensive but may provide a potential cure for an illness that currently requires lifelong care. The new treatments are not without challenges, as they require intensive care, chemotherapy, and lengthy hospital stays. Additionally, many Black Americans with sickle cell disease may be hesitant to trust a healthcare system that has historically failed to provide adequate care for the disease. However, for doctors and parents who have witnessed the suffering caused by the disease, the potential for a cure brings excitement and hope.

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