Finding cures for children's genetic diseases


NSW Funding for new viral vector manufacturing facility


Children’s Medical Research Institute thanks and congratulates the NSW Government for its vision in announcing $25 million in funding for a new advanced viral vector manufacturing facility at Westmead, to produce specialised gene therapy tools at clinical grade.

This investment reaffirms the NSW Government’s commitment to world-leading research conducted at the Westmead Precinct, and most importantly, toward transforming children’s lives.
Almost 25 years ago, Children’s Medical Research Institute teamed up with The Children’s Hospital at Westmead to establish a gene therapy research program. This long-term investment in research has helped to 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.
A vector is a microscopic tool used to deliver healthy copies of genes to tissues and organs within patients or deliver the ability to correct the genetic error at its source. 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.
One facility can produce vector to treat illnesses impacting everything from those with life-threatening liver disease to children going blind, but it can be extremely costly to get it to Australia and the waiting list is long.
Professor Ian Alexander, Head of the Gene Therapy Research Unit at Children’s Medical Research Institute, a 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 and across Australia.
“We see it as the beginning of something much greater,” he 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 specializes 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.”
“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 at Children’s Medical Research Institute 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.