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

Cell and Gene Therapies for Children

There are currently three 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

CHOP led the clinical trials of the first cell therapy to be approved by the FDA and has treated more patients than any other pediatric institution.
About Kymriah

Inherited Blindness

A one-time gene therapy product for children with a type of retinal dystrophy has its genesis in more than 10 years of research conducted at CHOP.
About Luxturna

Spinal Muscular Atrophy

CHOP was one of several institutions that participated in clinical trials of this FDA-approved gene therapy to treat SMA.
About Zolgensma

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.

Our Research

Diseases of Focus

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.