CHOP has consistently been at the forefront of clinical care and scientific discovery, with a focus on delivering better outcomes. Our physicians are pioneers and trailblazers with deep multidisciplinary expertise, and our 45 pediatric subspecialties are nationally and internationally recognized, with all our programs ranked, year after year. From precision diagnostics to novel therapeutics to world-class care that places families at the center, our uniquely comprehensive bench-to-bedside approach has led to countless breakthroughs and saved countless children’s lives.
A Lifesaving Heart Surgery Before Birth
Experts from CHOP performed lifesaving fetal open heart surgery to remove a tumor growing on Arley’s heart.
A Never-Before-Seen Disease, Diagnosed
CHOP doctors unlock the solution to Elijah’s mysterious illness when they identify a new autoimmune disease.
Changing the trajectory of children’s health
The future of pediatric medicine depends on breakthroughs at the smallest level: by treating diseases with cell and gene therapy. As pioneers of the first FDA-approved cell and gene therapies for children, CHOP is uniquely positioned to bring innovations from our labs quickly and safely to the bedside. Of the more than a dozen cell and gene therapies for pediatric conditions, many were developed with key CHOP contributions. Our teams continue to develop more options for children. CHOP is also a site or sponsor for 47 active clinical trials for cell and gene therapies, each with the potential to tangibly impact the trajectory of children’s health around the world. And CHOP is also committed to creating equitable access to cell and gene therapies for all families that turn to us for care.
First FDA-approved Cell Therapy
CHOP was the first hospital to use cell therapy to treat a child’s cancer. Today at CHOP, more than 500 patients – more than at any other hospital – receive a unique immunotherapy in which their own modified cells fight cancer.
First FDA-approved Gene Therapy for a Genetic Disease
Thanks to a CHOP-developed gene therapy for a devasting retinol disease, a little girl diagnosed with genetic blindness has her sight restored.
Gene Therapy for Sickle Cell Disease
Sickle cell disease is an inherited blood disorder that often causes debilitating pain and life-threatening complications. Many patients with the disease make dozens of ER visits a year, and must receive regular blood transfusions. And though recent breakthroughs in gene therapy are giving patients with sickle cell new hope – and new options – accessing these therapies can be challenging. In this episode of Breaking Through, cell and gene therapy expert Dr. Stephan Grupp and pediatric hematologist Dr. Alexis Thompson join Madeline to talk about how gene therapy for sickle cell disease can transform patients’ lives. They also discuss what teams at Children’s Hospital of Philadelphia (CHOP) are doing to ensure that these innovative therapies reach the children who need them most.
FDA Approves Two Gene Therapies for Sickle Cell Disease
CHOP has been on the leading-edge of treatment for sickle cell disease – an excruciating illness with no cure – for over a decade. A groundbreaking gene therapy finally provides hope.
A culture of innovation
From our expansive physical footprint – including our Care Network, inpatient locations, community hospital affiliations, vast research space and facilities dedicated to treating mental health – to research partnerships that span the globe, we channel resources into innovative spaces and initiatives that elevate the standards of care and encourage an environment of collaboration and discovery. Our 25+ CHOP-funded Frontier Programs are pioneering solutions for complex medical conditions often not available anywhere else in the world; eight are relentlessly searching for curative therapies. These discoveries wouldn’t be possible without our rich bank of biospecimens – the largest of its kind. These biological samples of pediatric DNA will unlock new treatments and cures to save more children’s lives.
Coming in 2025 with 350K+ sq. ft. of research space
Guns: The no. 1 killer of kids
CHOP not only treats children for trauma caused by the public health crisis of gun violence but has also launched innovative family education and gun lock distribution programs in the community.
The mental health crisis
The United States has never seen a mental health crisis like the one children and teens now face. CHOP is taking an innovative, comprehensive approach to address this crisis head on.
Equitable access to care
Together with legislators, educators, community leaders and families, we are removing barriers to care and finding innovative solutions to catalyze real, substantive changes for children and families.