Overview

• KJ was discharged after 307 days at Children’s Hospital of Philadelphia following two tailored CRISPR base-editing infusions delivered at seven and eight months of age.
• Within seven weeks of his second infusion, KJ began tolerating increased dietary protein and required lower doses of nitrogen-scavenger medications.
• An unprecedented six-month collaboration among CHOP, the University of Pennsylvania, Innovative Genomics Institute and Danaher accelerated therapy development while the FDA approved it within one week.
• KJ’s case was documented in the New England Journal of Medicine and showcased at the American Society for Gene and Cell Therapy.
• Clinicians will monitor long-term safety and aim to establish a scalable gene-editing platform for other rare genetic disorders.