Gene and cell therapies to be made in NSW

August 21, 2018

NSW Health has announced $2M of funding towards infrastructure supporting the production of gene and cell therapies in NSW. The project will be coordinated by Paediatrio, and led by researchers at Sydney Children’s Hospitals Network (SCHN) and the Children’s Medical Research Institute (CMRI).


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 to manufacture an essential but limited component of gene and cell therapies – the gene transfer vector.


Gene transfer vectors act like a Trojan horse to transport a gene into the existing DNA of a cell, and must therefore be made according to the highest regulatory standards of the Code of Good Manufacturing Practice (cGMP).


The leading team of investigators 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 Cell Medicine Facility and chief investigator 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 are


a, 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, which will be established 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. This NSW Health funding will boost grant support provided by The Kid’s Cancer Project for this trial, in line with their mission to provide improved cancer treatments for children across Australia.


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.  


Cleanroom facilities at the Gene and Cell Therapy Facility, Kids Research

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