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Cell and Gene Therapy Innovations

Cell and Gene Therapy Innovations

Little blonde girl smiling at camera
The future of improving children’s health depends on making breakthroughs at the microscopic level: by treating diseases with cell and gene therapy.

Across Children’s Hospital of Philadelphia (CHOP) — in multiple departments and laboratories — we are committed to discovering the next life-changing treatments. As pioneers of the first FDA-approved gene and cell therapies for children, we bring solutions from our labs to the bedside quickly and safely — so children with severe illnesses can reach their full potential. 

80+
faculty

More than 80 faculty engaged in research 

20+
programs

More than 20 unique clinical and pre-clinical programs

45+
clinical trials

More than 45 active clinical trials 

FDA approves two gene therapies for sickle cell disease

The Food and Drug Administration (FDA) has approved CASGEVY™ (exagamglogene autotemcel) and LYFGENIA™ (lovotibeglogene autotemcel), the first two gene therapies for the treatment of sickle cell disease in patients 12 years and older with recurrent vaso-occlusive crises (VOCs). 

FDA-approved cell & gene therapies

There are currently several FDA-approved cell and gene therapies available for use in children in the United States. Two of them were developed by experts at Children's Hospital of Philadelphia. 

Diseases of focus

Diseases and disorders with known and potential targets for cell and gene therapies are the focus of research in numerous departments and divisions across CHOP. Conditions with active research, clinical trials and treatments include: 

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Success Stories

Children’s Hospital of Philadelphia now offers the first-ever gene therapy treatment for Duchenne Muscular Dystrophy (DMD) to patients like 6-year-old Cash. This breakthrough treatment slows the progression of symptoms and can improve strength and endurance for patients with DMD.

Clinical programs offering cell & gene therapies

Children facing serious illness for which cell and gene therapies are available are cared for in the CHOP program that can best treat their specific illness. These include: 

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Cell & gene therapy supportive programs

The path from an idea in the lab to a clinical trial and finally to an FDA-approved therapy winds through these support programs. It’s with the expertise of these programs that discoveries are ushered through the complicated but critical steps toward safe and effective treatments. 

Lab worker sitting at microscope and smiling

Our team

The members of our team are global leaders in cell and gene therapy, bringing visionary research from the lab to the bedside and transforming the future of pediatric medicine. 

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