American doctors created and applied a personalized drug for the first time in the world using gene-editing technology specifically for an infant with a rare disease. The baby had a mutation in CPS1, which prevented the body from removing ammonia. Without treatment, he would have faced a liver transplant or lifelong dependence on medical equipment.
Within a record seven months, a team of 45 specialists developed the drug using “base editing,” a gentler version of CRISPR that changes individual DNA “letters” without making cuts. This allows the underlying cause of the disease to be addressed without damaging the rest of the genome. After three doses of treatment, no complications were reported, and the child shows improvements.
Most of the work was done for free. Developing a drug for one patient is not economically beneficial, so the project became an act of professional selflessness and a test of new opportunities in medicine. Programs in the United States and Europe are already underway to standardize and reduce the cost of personalized interventions—because individualized therapy can be a way to return to normal life without the risk of rejection and lifelong reliance on medications.
