About the Cell and Gene Therapy Collaborative
The Cell and Gene Therapy Collaborative (CGTC) at Children's Hospital of Philadelphia (CHOP) aims to significantly increase the volume of new pediatric cell and gene therapy research and to accelerate the pace of clinical development. The goal: develop new treatments for diseases that currently have few or no effective therapies.
Contributors to this important work include the Raymond G. Perelman Center for Cellular and Molecular Therapeutics, the Cellular Therapy and Transplant Section in the Division of Oncology, the Department of Pathology and Laboratory Medicine, the Division of Human Genetics, the Center for Applied Genomics, the Center for Fetal Research and faculty members from research and clinical programs across the institution. By connecting unique resources with world-class talent, CHOP will streamline the process of bringing cell and gene therapy research from the bench to our patients.
The Cell and Gene Therapy Collaborative takes a three-part approach:
- Advance new ideas by providing significant support to current faculty and recruiting new faculty in these fields
- Refine the existing operating model to enable more clinical trial activity
- Connect multiple groups involved in the development of cell and gene therapies to advance research efficiently
Our longstanding commitment of being at the forefront of cell and gene therapy approaches has already led to game-changing success. The world took notice when, in August 2017, the U.S. Food and Drug Administration approved chimeric antigen receptor (CAR) T-cell therapy for the treatment of young patients battling aggressive blood cancer. The CHOP Research Institute also played a vital role in developing a breakthrough one-time gene therapy for the treatment of vision loss due to a form of inherited retinal dystrophy, which received FDA approval in December 2017.
These innovative contributions by CHOP researchers, clinicians and collaborators heralded a new era of pediatric therapies that the Cell and Gene Therapy Collaborative will continue to foster.