World First: Baby Treated with Personalized CRISPR Gene Therapy is Now Thriving
A baby boy with a rare and fatal genetic disorder became the first person to receive a personalized CRISPR-based gene therapy, and is now thriving months after treatment.
HealthGNGV Editorial Team5 min readLast reviewed September 28, 2026
Good News Network reports on a groundbreaking medical achievement: a baby boy named KJ became the first person ever to receive a personalized CRISPR-based gene therapy. Born with carbamoyl phosphate synthetase 1 deficiency (CPS1), a rare metabolic disorder that prevents the body from processing nitrogen, KJ faced a grim prognosis.
Doctors at Children's Hospital of Philadelphia developed a custom treatment in record time. The therapy uses CRISPR technology to correct the genetic defect in his liver cells. Months after treatment, KJ is thriving.
