Cell and Gene Therapy Innovations
The future of improving children’s health depends on finding breakthroughs at the smallest, microscopic level: by treating diseases with cell and gene therapy.
Across Children’s Hospital of Philadelphia — in multiple departments and laboratories — we are committed to discovering the next life-changing treatments. Our proven track record as pioneers of the first FDA-approved gene and cell therapies for children positions us to bring innovations from our labs quickly and safely to the bedside, so children with severe illnesses can reach their full potential.
80+
faculty engaged in research
20+
unique clinical and pre-clinical programs
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:
- Cancer
- Neurological Disorders
- Hematological Disorders
- Ophthalmological Disorders
- Auditory Disorders
- Immunological/Inflammatory Disorders
- Metabolic Disorders
- Lysosomal Storage Disorders
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:
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.
- Clinical In Vivo Gene Therapy
- Cell & Gene Therapy Collaborative
- Cell & Gene Therapy Laboratory
- Cell & Gene Therapy Informatics
- Raymond G. Perelman Center for Cellular and Molecular Therapeutics which includes:
- Clinical Vector Core
- Research Vector Core
- Human Pluripotent Stem Cell Core
- Comparative Medicine Services Core
- Engineered Cell Therapy Core