Researchers and clinicians from the University of Sydney, Children’s Medical Research Institute and the Children’s Hospital at Westmead are the proud recipients of a $25 million NSW Government funding initiative that will see the state lead the way in gene and cell therapy.
The investment, announced yesterday by the NSW Minister for Health and Medical Research Brad Hazzard, will fund a new Good Manufacturing Practice facility at Westmead, to produce specialised gene therapy tools at clinical grade.
This investment reaffirms the NSW Government’s commitment to world-leading research at the Westmead Precinct – a partnership between the University, The Children’s Hospital at Westmead and Westmead Hospital – and, most importantly, toward transforming the lives of children with cancer and genetic diseases.
Almost 25 years ago, Children’s Medical Research Institute, which is an affiliate of the University of Sydney, teamed up with the Children’s Hospital at Westmead to establish a gene therapy research program. This long-term investment in research has helped put NSW in the position where it can now create a facility to manufacture clinical-grade vectors for gene therapy applications in paediatric and adult patients.
These developments will strengthen crucial collaborations in the Precinct – from design to distribution.
A vector is a microscopic carrier of pieces of DNA. It is used to deliver healthy copies of genes to tissues and organs within patients or deliver the ability to correct the genetic errors. While the technology is moving rapidly, production of vectors is not.
NSW, and in particular the Westmead Precinct, is already at the forefront of international gene therapy research. The aim of this project is to speed up research and translate it into cures for serious genetic diseases affecting children.
The facility will produce vectors to treat illnesses impacting everything from those with life-threatening liver disease to children going blind. Currently the vectors need to imported and its extremely costly to get them to Australia.
Professor Ian Alexander, Head of the Gene Therapy Research Unit at Children’s Medical Research Institute, senior clinician at The Children’s Hospital at Westmead and Professor of Paediatric and Molecular Medicine at the University of Sydney, said the manufacturing facility would be a boost to translation of academic research in NSW.
“We see it as the beginning of something much greater,” Professor Alexander said.
“It is about moving technology into the clinic, which, in future, will benefit many more patients by offering new and better treatment opportunities. This technology could translate into saving the lives of infants with life-threatening conditions.’’
Dr Leszek Lisowski heads the Translational Vectorology Group at CMRI and is Conjoint Senior Lecturer at the University of Sydney. His team will play a key role in the new facility, through training of staff and developing the manufacturing processes that will underpin operations. In addition, his team specialises in the development of novel vectors optimised for clinical applications targeting liver, eye and many other clinically important organs and tissues.
Dr Lisowski said that this new facility will allow Australian investigators to get around the "bottleneck" of getting vectors from overseas.
“The biggest bottleneck that slows down translation of gene therapy tools to the patient is a global lack of vector manufacturing capacity, which significantly extends the timeline and increases the cost of translational studies," he said.
“This facility will give Australian researchers prioritised and cost-effective access to clinical gene therapy reagents and will facilitate translation of a large number of exciting preclinical programs from bench to bedside.”
The team is excited by this vital investment and looks forward to partnering with government and other funders to enable the facility to achieve its full potential.
The Westmead Precinct is one of the largest health, education, research and training precincts in Australia and a key provider of jobs for the greater Parramatta and western Sydney region. Spanning 75 hectares, the Precinct includes four hospitals, four world-leading medical research institutes, two multidisciplinary university campuses and the largest research-intensive pathology service in NSW.
The University of Sydney has long been a proud partner of the Precinct and is in negotiations about developing a second major campus in the area. By 2050, that campus will include 25,000 students; 1000 staff and researchers; generate $21.7 billion for the NSW economy and support up to 20,000 jobs.
University of Sydney Vice-Chancellor and Principal Dr Michael Spence said that as part of our collaborative work in building a western Sydney global centre of excellence, Precinct partners are growing Australia’s advanced manufacturing capability.
“These developments will strengthen crucial collaborations in the Precinct – from R&D and design to distribution – in areas such as prevention and wellbeing, biomedical engineering, AI and personalised medicine,” Dr Spence said.
Faculty of Medicine and Health Executive Dean Professor Robyn Ward said: “This technology will scale up gene therapy using viral vectors from single-condition, life changing successes, for example in spinal muscle atrophy, to a national service.
“We are so proud of this leadership at the Westmead Precinct and with our health partners. It is a whole-of-lifespan, true bench-to-clinic approach."