A Leader in Cell and Gene Therapy

Julius, Cure Sickle Cell ambassador Children’s Hospital of Philadelphia’s (CHOP) cutting-edge advances in the field of cell and gene therapy have helped transform Philadelphia into Cellicon Valley. Because of the extraordinary success of pediatric studies led by CHOP, CAR T became the first-ever FDA-approved cell therapy. Now, there are already four FDA-approved CAR T products, and we are entering full speed into the era of cell and gene therapies — with CHOP’s work at the forefront, as detailed here.

Our Highlighted Programs and Services

Cellular Therapy and Transplant Section: This program consists of a preeminent team of specialists in immunotherapy (IP) and blood and marrow transplant (BMT). The program offers the most cutting-edge treatment options for a variety of severe illnesses, various cancers, and disorders of the blood and immune system.

Cell and Gene Therapy Collaborative: The Collaborative is designed to significantly increase the volume of new pediatric cell and gene therapy research and to accelerate the pace of clinical development by coordinating the efforts of multiple groups operating across the institution.

Sickle Cell and Red Cell Disorders Curative Therapy Center (CuRED): The Center offers integrated and coordinated care from multiple pediatric specialists for children with sickle cell disease, thalassemia, and other red cell disorders, and a robust research program that is on the cutting edge of the field.

Pediatric Comprehensive Bone Marrow Failure Center: This is a comprehensive, multidisciplinary center dedicated to providing world-class care for children and adults with inherited and acquired forms of bone marrow failure syndromes, which are diagnostically and therapeutically challenging.

Program for Integrated Immunodeficiency and Cellular Therapy (PIICT): This program provides integrated and comprehensive long-term care to children with primary immune deficiencies and functional immune abnormalities following cellular therapies such as hematopoietic stem cell transplant for specific types of leukemia.

Leukodystrophy Center: This Center provides comprehensive clinical care, diagnostic testing and the most advanced treatments available to infants, children and adolescents living with inherited disorders of the white matter of the brain and spinal cord.

CHOP’s Most Innovative Programs: Highlights of CHOP’s programs that are accelerating breakthroughs and solving the unsolvable.

Gene replacement therapy for spinal muscular atrophy: CHOP offers this as a treatment option for children who meet certain criteria.

CHOP’s Cell and Gene Therapy Clinical Trials

Upcoming Events

Dates for our continuing professional webinar series coming soon!

Resources for Professionals

CHOP Open: A library of online courses related to hematology and oncology.

“Breaking Through with Madeline Bell” podcast: CHOP President and CEO Bell interviews top CHOP clinicians and researchers, including episodes featuring Stephan Grupp, MD, PhD, Beverly Davidson, PhD, and Stefano Rivella, PhD.

Pediatric Cancer Immunotherapy/Bone Marrow Transplant Fellowship: CHOP and the University of Pennsylvania offer a one-year fellowship for qualified physicians who have completed three years of pediatric hematology/oncology fellowship training and who wish to pursue further training in cellular therapy and transplant.

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News and Articles

CHOP Researchers Develop Potentially Safer, More Effective Gene Therapy Vector for Blood Disorders

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