The FDA has approved the first-ever CRISPR-based medicine, called Casgevy, for the treatment of sickle cell disease. Developed by Vertex Pharmaceuticals and CRISPR Therapeutics, Casgevy has shown the potential to eliminate recurrent episodes of debilitating pain caused by sickle cell. The therapy works by precisely repairing DNA mutations, offering a potential long-term cure for the disease. However, the treatment is not without its challenges. Patients must undergo weeks of hospitalization and preparatory steps that can cause severe side effects. Additionally, the high price of Casgevy and the complexities of the treatment process may create barriers to reimbursement and access for patients.
https://www.statnews.com/2023/12/08/fda-approves-casgevy-crispr-based-medicine-for-treatment-of-sickle-cell-disease/