In December 2023, the FDA approved two novel treatments for Sickle Cell Disease, Casgevy and Lyfgenia, which use gene therapy to replace the faulty gene causing misshapen red blood cells. Sickle cell disease is a genetic condition affecting millions globally and predominantly in Sub-Saharan Africa and among non-Hispanic Black or African American populations in the US. These new treatments offer hope for better management of the disease, with Casgevy using CRISPR/Cas 9 gene editing to reactivate healthy fetal haemoglobin and Lyfgenia using a virus to paste a gene for producing normal haemoglobin. Clinical trials have shown promising results, although costs remain a concern for wider accessibility.
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FDA Approval for Sickle Cell Disease Gene-Therapy
