Children's Hospital of Philadelphia Marks One-Year Anniversary of World's First Personalized CRISPR Gene Therapy for Child with Rare Genetic Disease

PHILADELPHIA, Feb. 25, 2026 /PRNewswire/ -- 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...