Cell and Gene Therapy Collaborative
The Cell and Gene Therapy Collaborative aims to build on Children's Hospital of Philadelphia's longstanding position at the forefront of cell and gene therapy research, which has already led to game-changing success. The Collaborative is designed to significantly increase the volume of new pediatric cell and gene therapy research and to accelerate the pace of clinical development by coordinating the efforts of multiple groups operating across the institution.
Thinking Bigger: Transforming the Future of Healthcare
Learn how our world-renowned teams are solving unsolvable problems and accelerating breakthroughs in cell and gene therapy through our unique culture of innovation.
Cell and Gene Therapies for Children
There are currently six 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.
Acute Lymphoblastic Leukemia
Inherited Blindness
Spinal Muscular Atrophy
Beta Thalassemia
Hemophilia B
Cerebral Adrenoleukodystrophy
Clinical Trials
Innovation in the field of cell and gene therapy is moving at an incredible pace at CHOP. There are currently more than 20 clinical trials enrolling patients, with many more on the horizon.
Diseases of Focus
- Acute Lymphoblastic Leukemia
- Acute Myelogenous Leukemia
- Beta Thalassemia
- Choroideremia
- Hemophilia
- Leber Congenital Amaurosis
- Methylmalonic Acidemia
- MPS I
- MPS II
- Non-Hodgkin Lymphoma
- Phenylketonurias
- Sickle Cell Disease
- Spinal Muscular Atrophy
Our Programs and Services
Our Team
Our executive committee and principal investigators represent the many programs throughout the institution that support the development of new cell and gene therapies.