Establishment of small-scale cGMP vector manufacturing for gene and cell therapy clinical trials – laying the foundations for national large scale capacity

Project Overview

Gene therapy, a promising field using genes as medicine, holds vast potential for treating challenging genetic diseases and cancers that were previously considered untreatable. It involves introducing genetic material into cells using viral vectors as carriers to restore normal cell function.

The growing interest in gene therapy has created a significant demand for viral vectors suitable for clinical trials. This program has established a vector production facility that adheres to good manufacturing practices (cGMP) standards to meet this rising demand for use in human clinical trials. This achievement places New South Wales at the forefront of vector production in Australia, offering economic benefits through intellectual property rights and commercialisation.

This facility leverages our world-class expertise in vector development and enables the initiation of crucial paediatric gene therapy clinical trials for a range of diseases such as cancer, eye, kidney and liver conditions. Additionally, it can produce vectors for the broader New South Wales and Australian research community, promoting the advancement of gene therapy for clinical applications. This transformative initiative has also set the foundation for establishing a large-scale national production capacity with global reach.


This project established cGMP-grade viral vector manufacturing in New South Wales for quick deployment in paediatric gene therapy trials, targeting cancer and rare genetic diseases.

Lead Investigators

  • Ian_Alexander@2x

    Professor Ian Alexander

    Head, Gene Therapy Unit, Children’s Medical Research Institute


  • Dr Belinda Kramer

    Co-Group Leader, Cancer Gene Therapy Group, Children’s Cancer Research Unit, Sydney Children’s Hospitals Network
  • Professor Stephen Alexander

    Head, Paediatric Nephrology, Sydney Children’s Hospitals Network
  • Professor Robyn Jamieson

    Head, Discipline of Genetic Medicine, Sydney Children’s Hospitals Network & Head, Eye Genetics Research Unit, Children’s Medical Research Institute
  • Dr Geoff McCowage

    Group Leader, Cancer Gene Therapy Group, Children’s Cancer Research Unit, Sydney Children’s Hospitals Network
  • Dr Leszek Lisowski

    Group Leader, Translational Vectorology, Children’s Medical Research Institute & Manager, Vector and Genome Engineering Facility, Children’s Medical Research Institute
  • Professor John Rasko

    Director, Cell and Molecular Therapies, Royal Prince Alfred Hospital & Head, Gene and Stem Cell Therapy Program, Centenary Institute