Medical Marvels

New medicines to treat sickle-cell disease and beta thalassaemia, two genetic blood disorders, will make headlines in 2024. Most notable of these is the first CRISPR-gene-edited drug, which made its historic arrival in late 2023. Gene editing uses molecular scissors to edit DNA. It is a more precise form of modification than gene therapy, an older technology that uses a viral vector to inject a working gene into a cell. Gene editing has moved astonishingly quickly through drug pipelines – much faster than gene therapies, which have been slow and difficult to develop.