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Abstract
Gene and cell therapy hold transformative potential for treating various illnesses by modifying or replacing genetic material or cells. In 2023, significant strides were made in the approval of new gene and cell therapy products, notably Lyfgenia, a gene therapy for sickle cell disease. Using a modified virus, Lyfgenia introduces a functional beta-globin gene into the patient's blood stem cells. This approval, alongside advancements in precise gene-editing techniques like CRISPR-Cas9, signifies substantial progress in correcting genetic abnormalities. Moreover, novel cell therapy products, including chimeric antigen receptor-like T cell (CAR-T) treatments for cancer, gained approval in 2023. These breakthroughs mark a transformative era in medicine, with the potential to enhance millions of lives and revolutionize medical practices.
