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.

faculty engaged in research
unique clinical and pre-clinical programs
active clinical trials

FDA Approves Two Gene Therapies for Sickle Cell Disease

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.

  • Hemgenix
  • Skysona
  • Vyjuvek
  • Casgevy
  • Lyfgenia
  • Lenmeldy

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:

  • 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.

Our Team