Cell and Gene Therapy Innovations
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.
More than 80 faculty engaged in research
More than 20 unique clinical and pre-clinical programs
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.
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.
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.
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.