In 2024, gene therapy has made significant strides, particularly in treating rare genetic disorders. Leading companies like CRISPR Therapeutics and Bluebird Bio are at the forefront, developing transformative therapies that can edit faulty genes directly. This has opened new treatment avenues for conditions such as sickle cell disease and β-thalassemia, which previously had limited treatment options. Casgevy, a CRISPR-based therapy, has received approval, marking a major milestone in the use of gene editing for life-altering treatments
However, there are still challenges ahead. Variations in genetic makeup across populations make designing universal therapies difficult, as different mutations can cause the same disease. Despite these hurdles, the future of gene therapy looks promising, especially for blood-based disorders, where gene-editing techniques can be applied more easily