The first CRISPR therapy
A gene-editing cure for sickle cell disease is approved, eleven years after the tool was invented.
- When
- Known to
- ± 12 hours
- Era
- Contemporary, Holocene
Casgevy was authorised in the United Kingdom on 16 November 2023 and by the US FDA on 8 December, treating sickle cell disease and beta thalassaemia by editing a patient's own stem cells to switch fetal haemoglobin back on. It is the first approved medicine based on CRISPR, a technique first described in 2012. The price — over two million dollars — leaves it out of reach in the countries where sickle cell is most common.