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Developing CAR T cells and gene viral vectors rapidly for paediatric trials for cancer, eye and kidney disease

Sydney Children's Hospital Network

  • Cell and Gene Therapy Grants
Chief Investigator/s:
  • Professor Ian Alexander

Gene therapy, or the use of genes as medicine, has immense therapeutic potential in the treatment of many genetic diseases and cancers that are currently difficult or impossible to treat. While progress in the development of the underpinning gene transfer technology (vectors) has been hard won, we are finally witnessing astounding successes in human clinical trials in both children and adults.

This in turn has led to a global upsurge in clinical trial activity for a vast number of troubling diseases, and unprecedented demand for gene transfer vectors prepared to the exacting GMP manufacturing standards required for human clinical use. This demand has dramatically out-stripped supply and has created a clinical trial log-jam that threatens to slow the realisation of therapeutic benefit to the many potential beneficiaries of gene therapy.

This project will develop CAR T cells to target a broad range of paediatric tumours, and CAR T cells to protect kidney transplants from chronic allograft nephropathy, as well as gene viral vectors to target blinding eye disease and to treat childhood kidney disease. This will allow us to quickly initiate desperately needed paediatric trials for cancer, eye and kidney disease.