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Gene therapy takes a giant leap forward as Exa-cel becomes the world’s first CRISPR/Cas9 gene-edited therapy approved to treat sickle cell disease and thalassemia. ....
5.2% of the global population carry hemoglobin abnormalities, resulting in 300,000 to 400,000 children born with severe hemoglobinopathies annually. ....
Casgevy is the first medicine to be licensed that uses the gene-editing tool CRISPR, which won its inventors the Nobel Prize in 2020, Britain’s medical regulator says ....
Research explores the advancements, challenges, and economic implications of gene therapy for various diseases, including inherited blood disorders, malignancies treated with CAR-T cells, and diseases targeted by in vivo AAV vectors. The review highlights the transformative potential of gene therapies while acknowledging the complexities in manufacturing, safety, efficacy, and long-term durability. ....