The approval of Casgevy, the new CRISPR option for sickle cell disease, is big news for American patients. The list price is high ($2.2 million) although lower than the non-CRISPR gene therapy approved by the FDA for the same pathology the same day. But in addition to economic sustainability, another issue worries scientists, clinicians, and patients: infertility.
The progress of the new therapies of the CRISPR era can be told by interweaving two stories. The first is the one featuring Victoria, Carlene, Patrick, Alyssa, Terry and many others. There are over two hundred patients who have so far undergone some experimental treatment based on genome editing, i.e. the targeted correction of DNA instead of the addition of extra genes as in classical gene therapy. These women and men suffering from serious diseases had to face increasing pain and sacrifice until they decided to pin their hopes on a new type of experimental therapy, which is promising but not without risks. For the unluckiest of them, this act of courage and faith in science was not enough, but for many of these pioneers, life really did change. In fact, there are already dozens of people who have managed to free themselves (hopefully in the long term) from the burden of a rare genetic disease or, in some cases, leukaemia. Along with genetically edited cells, a new normalcy has arrived for them and the chance to finally think about the future.