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

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 (viral vectors) has been hard won, we are now 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 current good manufacturing practices (cGMP) standards required for human clinical use. This demand out-stripped supply of clinical-grade vectors and created a clinical trial log-jam that threatens to slow down the realisation of therapeutic benefits to the many potential beneficiaries of gene therapy.

To address this issue, this project sought to establish a small-scale clinical grade vector manufacturing capacity in NSW. This directly builds on our world-class expertise in vector development and will allow for the urgently needed paediatric trials for cancer, eye and kidney disease. NSW capacity for gene therapy vectors has been increased through this project with clinical grade LV gene therapy vector production significantly progressed. The $2 million project provided the groundwork for clinical grade LV and rAAV gene therapy vector production.

This initial funding has been enhanced and expanded through an additional $134 million in NSW Government funding to achieve a comprehensive vision of a commercial-scale GMP viral vector manufacturing facility now under construction.

This facility will in turn increase the number of early phase gene and cell therapy clinical trials initiated in NSW and Australia that arise from pre-clinical data generated within the academic sector. This can be demonstrated with the recent MRFF Clinical Trials Activity grant success of $2.3 million to deliver a gene therapy clinical trial activity at SCHN in February 2023, specifically The E2CAR Trial: A Phase I clinical trial to evaluate administration of EphA2 targeted CAR T cells to children with sarcoma.

This investment has put in place the necessary infrastructure to deliver a national large-scale production capacity to meet the growing demand for viral vectors for use in gene therapy research and clinical trials in Australia and internationally.

Professor Ian Alexander
Sydney Children’s Hospitals Network, Principal Researcher

• Dr Belinda Kramer, Sydney Children’s Hospitals Network
• Professor Stephen Alexander, Sydney Children’s Hospitals Network
• Professor Robyn Jamieson, Sydney Children’s Hospitals Network
• Dr Geoff McCowage, Sydney Children’s Hospitals Network
• Dr Leszek Lisowski, Children’s Medical Research Institute
• Professor John Rasko, Royal Prince Alfred Hospital

NSW-Advanced-Therapeutics-Ecosystem-Prosp 12 cGMP

See Luminesce Alliance Publications – completed research – Establishment of small-scale cGMP vector manufacturing for gene and cell therapy clinical trials