Finding cures for children's genetic diseases

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Gene therapy collaboration rewarded

23/Aug/2018  

The NSW State Government has announced it will award $2 million in funding to support the production of gene and cell therapies by researchers at Sydney Children’s Hospitals Network (SCHN) and the Children’s Medical Research Institute (CMRI).


Gene therapy uses genes as medicine, which has immense therapeutic potential in the treatment of many genetic conditions and cancers that are currently difficult or impossible to treat.  In gene therapy for genetic conditions, a “faulty” gene is either replaced or edited, with the aim of restoring normal function to the patient.

The same technology can also be used to introduce new genes into immune cells, which allows them to trigger an immune response against disease-causing entities, such as tumour cells.



Global research efforts to test the safety and efficacy of gene and cell therapies in clinical trials are threatened by an increased demand for an essential and limited component – the gene transfer vector.

Gene transfer vectors act like a Trojan horse to transport a gene into the existing DNA of a patient’s cells.  
In the case of gene therapy for genetic conditions, the new or repaired gene is introduced to override or correct the instructions contained in the faulty gene.  In immune-based anti-cancer therapy, however, the new gene encodes for novel proteins that allow immune cells to recognise, bind to, and eliminate biological targets that cause disease.

Vector supply pressures are especially felt in an Australian context, as vectors are often commercially sourced from overseas at high financial cost and waiting times, which can be as long as two years. 

Necessarily complex manufacturing and regulatory conditions do not ease the supply burden for researchers, who often do not have the resources or expertise to manufacture in-house.  

Led by Professor Ian Alexander, clinician-scientist and head of the Gene Therapy Research Unit collaboration between Kids Research, SCHN, and CMRI, the team will establish capacity on-site at The Children’s Hospital at Westmead to manufacture gene transfer vectors according to the highest regulatory standards of the Code of Good Manufacturing Practice (cGMP). 

The leading team of investigators from Kids Research, CMRI, Royal Prince Alfred Hospital (RPAH) and the CSIRO have 200 collective years of research in genetic engineering and genetic medicine, and many were part of the first group in Australia to safely manufacture clinical grade vectors for use in an early phase clinical trial.

With essential infrastructure already in place, such as the cleanroom facilities of the Kids Research gene and celle medicine facility Dr Leszek Lisowski’s vector and genome engineering facility at CMRI, the team are well equipped to embark on the small scale cGMP clinical grade vector production initiative.



Harnessing their combined scientific, technical, clinical and regulatory expertise in this area, the team will extend the current capacity by introducing new technologies. This includes manufacturing vectors using a liquid suspension of cells, rather than cells that are fixed in a single layer on a plate, increasing flexibility and the possibility for up-scaling in the future. 

The establishment of such a capacity on-site at Westmead will enable researchers and clinicians to efficiently react to the needs of patients, accelerating the translation of innovative research from bench to bedside. 

Initially, the production will facilitate the rapid initiation of four paediatric trials for cancer, eye and kidney disease. Subject to receiving all ethics and regulatory approvals, the aim is for the first trial to begin in 2021. 

The intention is that the first trial will test a gene and cell-based therapeutic approach for paediatric solid tumours, where modified immune cells express a novel surface protein that can recognise tumour cells and attack them.

While local residents from NSW will be the first to benefit from timely access to innovative gene and cell therapies, the service is expected to lay the foundations for national, and even global, production capacity, improving the lives of people with rare diseases and cancer across Australia and beyond.   

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