Infant treated with personalized CRISPR gene editing therapy for CPS1 deficiency: Live Timeline (Feb 2026)

As of today, KJ continues to thrive, showing no adverse effects from the treatment, growing well, and meeting developmental milestones, though ongoing monitoring is planned for his lifetime.

Further analysis and reports continue to affirm the early success of the personalized CRISPR base-editing in treating CPS1 deficiency, underscoring its significance for genomic medicine.

News outlets widely report on the medical breakthrough, emphasizing the potential for personalized gene editing to treat other ultra-rare genetic diseases.

The research team presents KJ's case at the American Society of Gene & Cell Therapy Annual Meeting, highlighting the rapid development and administration of the personalized therapy.

The groundbreaking case study detailing KJ's successful treatment and initial outcomes is published in The New England Journal of Medicine.

KJ receives a third infusion of the personalized CRISPR therapy to further enhance the treatment's efficacy.

Early results show significant improvement in KJ's condition, allowing him to tolerate increased protein intake and reduce his reliance on ammonia-lowering medications.

KJ receives a second infusion of the personalized CRISPR therapy as part of the ongoing treatment protocol.

At approximately six months of age, KJ receives the first infusion of his customized CRISPR base-editing therapy, marking the world's first administration of such a personalized treatment.

Following the failure of conventional treatments and consideration of a liver transplant, a team at Children's Hospital of Philadelphia (CHOP) and Penn Medicine begins developing a personalized CRISPR base-editing therapy specifically for KJ.

Infant KJ is diagnosed with severe carbamoyl phosphate synthetase 1 (CPS1) deficiency, a rare and life-threatening metabolic disorder, just days after his birth.