Founded in 1855 as the first hospital in the United States dedicated entirely to pediatric care, Children’s Hospital of Philadelphia (CHOP) has helped define modern pediatric medicine for over 170 years. Operating in close academic partnership with the Perelman School of Medicine at the University of Pennsylvania, CHOP functions as one of the world’s most prolific pediatric research institutions and quaternary clinical referral centers.
Consistently ranked among the premier children’s hospitals in the nation by U.S. News & World Report, CHOP’s global reputation is built on groundbreaking achievements across three critical frontiers: living cell and gene therapies in Pediatric Oncology, pioneering in-utero interventions through the Richard D. Wood Jr. Center for Fetal Diagnosis and Treatment (CFDT), and translational clinical discoveries in Genomics and Individualized Medicine.
Core Specialty Overview
| Specialty Area | Key Distinctions | Advanced Interventions |
| Pediatric Oncology | Birthplace of CAR T-cell therapy (Kymriah) | Real-time in-OR sequencing & 3D tumor organoids |
| Fetal Medicine & Surgery | Leader of the landmark NIH MOMS trial | Open in-utero spina bifida repair & FETO procedures |
| Genomics & Rare Diseases | Rapid neonatal genome sequencing hub | First-in-human bespoke CRISPR gene-editing trials |
| Pediatric Cardiology | Comprehensive single-ventricle reconstruction | Fetal cardiac interventions & pediatric heart transplants |
Pediatric Oncology: The Birthplace of Cellular Immunotherapy
CHOP’s Cancer Center has led the global movement away from high-toxicity chemotherapies toward targeted, living cellular immunotherapies.
- The CAR T-Cell Revolution: In 2012, CHOP oncologists treated six-year-old Emily Whitehead, the first pediatric patient in the world to receive experimental Chimeric Antigen Receptor (CAR) T-cell therapy for relapsed acute lymphoblastic leukemia (ALL). This trial formed the basis for tisagenlecleucel (Kymriah), the first FDA-approved gene therapy.
- In-OR Rapid Sequencing: Utilizing rapid methylation profiling directly inside the operating suite, neurosurgeons receive real-time molecular fingerprints of brain tumors during active resections.
- Patient-Derived Organoids: Laboratory teams culture 3D mini-tumors from patient biopsy tissue within 1–2 weeks to test targeted drug sensitivities before administering therapies to the child.
- Targeted Radiotherapy: Advanced MIBG therapy suites deliver targeted internal radiation directly to high-risk neuroblastoma cells while keeping families close in radiation-shielded adjoining rooms.
World Leadership in Fetal Medicine & In-Utero Surgery
The Richard D. Wood Jr. Center for Fetal Diagnosis and Treatment (CFDT) at CHOP is one of the world’s highest-volume fetal health centers, offering diagnostic precision and surgical correction before birth.
- The Landmark MOMS Trial: CHOP was the lead clinical center for the NIH Management of Myelomeningocele Study (MOMS). The study proved that repairing spina bifida defects in the womb significantly reduces the need for brain shunts, reverses Chiari II malformations, and doubles a child’s chances of walking independently.
- Garbose Family Special Delivery Unit (SDU): The world’s first labor and delivery unit built inside a freestanding pediatric hospital exclusively for mothers carrying babies with prenatally diagnosed anomalies, eliminating the need for emergency newborn transit across town.
- Fetoscopic Interventions: Specialists perform minimally invasive fetoscopic laser photocoagulation for Twin-to-Twin Transfusion Syndrome (TTTS) and Fetoscopic Endoluminal Tracheal Occlusion (FETO) for severe congenital diaphragmatic hernia (CDH).
Groundbreaking Pediatric Genetics & Precision Medicine
CHOP houses one of the world’s most advanced pediatric genetics networks, translating fundamental genomic sequencing into tailored clinical interventions.
- Bespoke CRISPR Gene Editing: In a landmark clinical first, CHOP researchers designed and administered individualized, mutation-specific CRISPR gene editing to treat an infant with a fatal inborn error of metabolism (CPS1 deficiency).
- Baby Eagle Initiative: Provides ultra-rapid whole-genome sequencing for critically ill infants in the neonatal intensive care unit (NICU), delivering definitive genetic diagnoses within days.
- Center for Applied Genomics (CAG): One of the largest pediatric biorepositories globally, containing genomic data from over 100,000 children to match rare phenotypic traits with actionable targeted drugs.
Patient Navigation & Global Family Services
For families traveling from other states or internationally, CHOP provides coordinated care pathways to streamline appointments, records, and travel logistics.
| Patient Pathway | Key Steps & Clinical Actions |
| Intake & Triage | Secure digital upload of clinical records, prenatal imaging, and pathology slides. |
| Multidisciplinary Review | Sub-specialists assemble a consolidated multi-day schedule before arrival. |
| Global Family Support | Multilingual interpreters, medical visa letters, and dedicated family lounges. |
| Transition of Care | Post-discharge care coordination with local pediatricians and remote telehealth monitoring. |
Conclusion
Children’s Hospital of Philadelphia stands as a premier global institution in pediatric medicine through three core strengths:
- Pioneering Cellular Oncology: Developed the first FDA-approved CAR T-cell therapy (Kymriah), establishing living gene therapies for childhood leukemias.
- Fetal Medicine Authority: Established open fetal surgery protocols, led the NIH MOMS trial for prenatal spina bifida repair, and operates the Garbose Special Delivery Unit.
- Genomic Translation: Drives rapid neonatal whole-genome sequencing and first-in-human individualized CRISPR therapeutics for rare pediatric genetic conditions.
For families seeking definitive answers for complex prenatal diagnoses, treatment-resistant childhood malignancies, or undiagnosed genetic conditions, CHOP delivers academic pediatric medicine backed by pioneering research and clinical expertise.