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, or the use of genes as medicine, has immense therapeutic potential on the treatment of many difficult or impossible to treat genetic diseases and cancers. It involves the insertion of genetic material into a cell via a viral vector (a carrier), to restore the cell’s normal function.  The increasing interest in gene therapy has lead to a high demand for the production of viral vectors for use in clincial trials. This program sees the establishment of a vector production facility that meets good manufacturing practices (cGMP) standards to meet the increasing demand for the use in human clinical trails. It will allow New South Wales to be at the forefront of vector production in Australia and economic potential can be gained through intellectual property rights and commercialisation.

The facility will directly build on our world class expertise in vector development and will allow for urgently needed paediatric gene therapy clinical trails for cancer, eye and kidney disease to be initiated. The facility will also have the capacity to produce vectors for the broader New South Wales and Australian research community as a means to advance the use of gene therapy for clinical use. Projecting forward, this transformative initiative will form the foundations for establishing a large-scale national production capacity with a global outlook.


This project will establish a NSW-based cGMP grade manufacturing capacity for viral vectors for rapid deployment in paediatric gene therapy clinical trials targeting cancer and genetic rare disease

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