One year since infant KJ received world's first personalized CRISPR gene therapy for rare genetic disease.
On February 25, 2026, in Philadelphia, it has been one year since an infant named KJ, who was diagnosed with severe carbamoyl phosphate synthetase 1 (CPS1) deficiency, made history by becoming the first person worldwide to undergo a groundbreaking personalized CRISPR-based gene editing therapy. This innovative therapy was created specifically for KJ and marked a significant advancement in genetic treatment.