Children’s Hospital of Philadelphia Marks One-Year Anniversary of World’s First Personalized CRISPR Gene Therapy for Child with Rare Genetic Disease
February 25, 2026 marks one year since KJ, an infant born with severe carbamoyl phosphate synthetase 1 (CPS1) deficiency, became the world’s first person to receive a personalized CRISPR-based gene editing therapy. The therapy was developed by physician-scientists at Children’s Hospital of Philadelphia (CHOP) and Penn Medicine, and in May 2025, CHOP shared that the […]