Founded in 1855 as the first hospital in the United States dedicated exclusively to the care of children, 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 most prolific pediatric research institutions and quaternary clinical referral centers in the world.
Consistently ranked among the top children’s hospitals in the nation by U.S. News & World Report, CHOP’s global reputation is defined by groundbreaking achievements in three critical frontiers: 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 discoveries in Genomics and Individualized Medicine.
1. Transforming Pediatric Oncology: The Birthplace of Cellular Immunotherapy
CHOP’s Cancer Center has led the global shift away from high-toxicity chemotherapies toward targeted, living cellular immunotherapies.
CHOP Pediatric Oncology Engine
┌─────────────────────────┬─────────────────────────┬─────────────────────────┐
│ CAR T-Cell Cellular │ Precision Genomic │ Advanced Neuroblastoma │
│ Immunotherapy │ Oncology Trials │ & Solid Tumor Research │
├─────────────────────────┼─────────────────────────┼─────────────────────────┤
│ • 1st pediatric CAR-T │ • Real-time in-OR │ • Target identification │
│ patient (Emily W.) │ tumor sequencing │ for high-risk ALK & │
│ • Co-developed Kymriah │ • Lab-grown patient- │ MYCN gene alterations │
│ (tisagenlecleucel) │ derived organoids │ • MIBG radiotherapy │
└─────────────────────────┴─────────────────────────┴─────────────────────────┘
The CAR T-Cell Revolution (Kymriah)
In 2012, oncologists at CHOP, led by Dr. Stephan Grupp in collaboration with the University of Pennsylvania, 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/refractory acute lymphoblastic leukemia (ALL).
By genetically modifying a patient’s own T-cells to express a synthetic receptor targeting the CD19 protein on cancer cells, the therapy achieved complete remission. This landmark clinical trial formed the scientific foundation for tisagenlecleucel (Kymriah), which in 2017 became the first-ever gene therapy and CAR-T treatment approved by the U.S. FDA.
Intraoperative Sequencing & Patient-Derived Organoids
Through the Advanced Personalized Therapeutics and Precision Surgery Program in Childhood Cancers (Frontier Program), CHOP integrates precision molecular biology directly into the operating theater:
- In-OR Rapid Sequencing:Deploying long-read genome sequencing and methylation profiling directly in the operating suite, providing real-time molecular fingerprints of brain tumors to neurosurgeons while the patient is on the operating table.
- 3D Tumor Organoids:Culturing miniature 3D patient-derived tumor models in the laboratory within 1 to 2 weeks to test specific drug sensitivities before administering therapies to the child.
2. World Leaders in Fetal Medicine & In-Utero Surgery
The Richard D. Wood Jr. Center for Fetal Diagnosis and Treatment (CFDT) at CHOP is one of the oldest, highest-volume, and most comprehensive fetal health centers in the world, having evaluated tens of thousands of complex pregnancies.
CHOP In-Utero Care and Delivery Pathway
┌──────────────────────┐ ┌──────────────────────┐ ┌──────────────────────┐
│ Comprehensive Fetal │ ──> │ Multidisciplinary │ ──> │ Open Fetal Surgery / │
│ 3D Ultrasound & MRI │ │ Consensus Evaluation │ │ Fetoscopic Occlusion │
└──────────────────────┘ └──────────────────────┘ └──────────────────────┘
│
Surgical or
In-Utero Delivery
Pathway
│
▼
┌──────────────────────┐ ┌──────────────────────┐ ┌──────────────────────┐
│ Long-Term Pediatric │ <── │ Specialized Level IV │ <── │ Garbose Special │
│ Follow-Up Clinics │ │ N/IICU Care Teams │ │ Delivery Unit (SDU) │
└──────────────────────┘ └──────────────────────┘ └──────────────────────┘
The Landmark MOMS Trial for Spina Bifida
Under the leadership of pediatric surgeon Dr. N. Scott Adzick, CHOP was the clinical anchor and lead investigative center for the National Institutes of Health (NIH) Management of Myelomeningocele Study (MOMS).
Published in the New England Journal of Medicine, this landmark trial proved that surgically repairing spina bifida lesions while the fetus is still inside the womb significantly reduces the need for brain shunts, reverses hindbrain herniation (Chiari II malformation), and doubles the child’s chances of walking independently compared to standard post-birth repairs.
The Garbose Family Special Delivery Unit (SDU)
CHOP was the first pediatric hospital in the world to open a dedicated, specialized labor and delivery unit exclusively for mothers carrying babies with prenatally diagnosed birth defects:
- Mothers and high-risk infants remain together under one roof, eliminating the need for emergency newborn transport across cities.
- Features dedicated operating rooms engineered for immediate post-delivery neonatal stabilization, EXIT (Ex Utero Intrapartum Treatment) procedures, and emergency extracorporeal membrane oxygenation (ECMO).
Complex Fetal Interventions Performed:
- Congenital Diaphragmatic Hernia (CDH): Minimally invasive Fetoscopic Endoluminal Tracheal Occlusion (FETO) to promote in-utero lung growth.
- Twin-to-Twin Transfusion Syndrome (TTTS): Fetoscopic selective laser photocoagulation of shared placental blood vessels.
- Sacrococcygeal Teratoma (SCT) & Fetal Lung Lesions: Open in-utero resections to prevent high-output fetal cardiac failure.
3. Groundbreaking Genetics & Precision Medicine
CHOP houses one of the world’s most advanced pediatric genetics networks, translating fundamental genomic sequencing into personalized therapies for rare and orphan diseases.
Genomic Discovery to Bedside Care
┌──────────────────────┐ ┌──────────────────────┐
│ Rapid Whole-Genome │ ── Target Discovery>│ Bespoke CRISPR / │
│ Sequencing (Baby E.) │ │ In Vivo Gene Editing │
└──────────────────────┘ └──────────────────────┘
│
Curative Delivery
│
▼
┌──────────────────────┐ ┌──────────────────────┐
│ Lifelong Metabolic │ <── Bedside Care ── │ Roberts Individualized│
│ & Cellular Stability │ │ Genetics Diagnostics │
└──────────────────────┘ └──────────────────────┘
First-in-Human Bespoke CRISPR Gene Editing
In a historic clinical advance, CHOP and Penn Medicine researchers designed and administered the world’s first individualized CRISPR gene-editing therapy for an infant (Baby KJ) diagnosed with severe carbamoyl phosphate synthetase 1 (CPS1) deficiency—a fatal metabolic urea cycle disorder.
By creating a customized therapeutic engineered specifically for the child’s precise DNA mutation, clinicians successfully stabilized the metabolic crisis.
Roberts Individualized Medical Genetics Center (IMGC)
The IMGC serves as the central hub for precision diagnostics, evaluating thousands of children with unexplained neurodevelopmental, cardiovascular, and metabolic disorders:
- Rapid whole-genome sequencing in the neonatal intensive care unit (Baby Eagle Initiative) to provide definitive diagnoses within days.
- Integration with the Center for Applied Genomics (CAG), which maintains one of the world’s largest pediatric biobanks with genomic data from over 100,000 children.
4. Specialty Performance Snapshot
| Clinical Specialty | Benchmark Standing & National Highlights | Primary Clinical Strengths |
| Pediatric Cancer Care | Top-Ranked Nationally | Pioneers of CAR T-cell therapies, neuroblastoma clinical trials, precision oncology. |
| Fetal Medicine & Surgery | Pioneer & Highest Global Volume | MOMS trial leaders, open fetal surgery, Garbose Special Delivery Unit. |
| Genomics & Rare Diseases | World-Class Referral Hub | Custom CRISPR gene-editing, rapid whole-genome sequencing, metabolic therapies. |
| Pediatric Cardiology | Consistently Top 5 Nationally | Complex single-ventricle reconstructions, fetal cardiac interventions, heart transplants. |
| Pediatric Orthopedics | Top Tier Nationally | Thoracic insufficiency syndrome, VEPTR expandable rib implants, complex spine surgery. |
5. Family Support & Global Patient Navigation
Recognizing the emotional and logistical burdens placed on families of critically ill children, CHOP embeds comprehensive support into its care delivery model.
┌────────────────────────┐ ┌────────────────────────┐ ┌────────────────────────┐
│ Global Intake & Remote │ ───> │ Multidisciplinary Team │ ───> │ Integrated Bedside, │
│ Record Diagnostics │ │ Consult & Staging Plan │ │ SDU or Surgical Care │
└────────────────────────┘ └────────────────────────┘ └────────────────────────┘
- Global Patient Services: Coordinates medical visa documentation, multilingual medical interpreters, appointment consolidation, and long-term accommodation for families visiting from over 100 countries.
- Child Life & Creative Arts Therapies: Certified Child Life specialists, facility therapy dogs, and music/art therapists help children process hospital stays through age-appropriate preparation and play.
- The Connelly Resource Center for Families: An on-campus sanctuary offering private rest suites, laundry facilities, a medical library, and child-care assistance for parents staying at their child’s bedside.
Summary
Children’s Hospital of Philadelphia stands as a global leader in pediatric healthcare through three core achievements:
- Cancer Cellular Therapies: Co-developed the world’s first approved CAR T-cell gene therapy (Kymriah), transforming outcomes for pediatric leukemia.
- Fetal Medicine Authority:Established open fetal surgery protocols, led the landmark MOMS trial for spina bifida, and operates the Garbose Special Delivery Unit.
- Precision Genomic Medicine:Pioneers rapid neonatal genome sequencing and customized CRISPR gene-editing therapies for rare pediatric diseases.
For families facing complex prenatal anomalies, treatment-resistant childhood malignancies, or undiagnosed genetic disorders, CHOP delivers academic pediatric medicine backed by scientific discovery and specialized care.