Children’s Medical Research Institute welcomes the NSW Government’s recent announcement that it will leverage world-class cell and gene therapy expertise to establish Australia’s first commercial-scale viral vector manufacturing facility in the Westmead Health and Innovation District and is inviting partners to co-invest.

This new facility is critically important to the work of CMRI’s gene therapy programs, including those led by Professor Ian Alexander, Professor Robyn Jamieson, Associate Professor Anai Gonzalez Cordero, and Associate Professor Leszek Lisowski.

A vector is a microscopic tool used to deliver healthy copies of genes to patients’ tissues and organs, or to deliver the ability to correct the genetic error at its source. While the technology is moving rapidly, the ability to produce high-quality (clinical-grade) vectors has been a roadblock.

NSW, and in particular the Westmead precinct, is at the forefront of international gene therapy research. The availability of clinical-grade viral vector production capability in Australia, located next door to CMRI, will accelerate the ability to translate our research, into the clinic – as potential cures for serious genetic diseases affecting children.

Currently, there is a NSW-Government-funded pilot-scale viral vector facility in the Westmead Health and Innovation District which Professor Alexander and Associate Professor Lisowski helped to establish.

Last year, the NSW Government announced it would invest $25m to develop this capacity further and has now called on private biotech partners to co-invest in a larger, commercial-scale facility that will serve Australia and international markets.

Professor Ian Alexander, Head of the Gene Therapy Research Unit at CMRI, 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.’’

Associate Professor Gonzalez Cordero said she and Professor Jamieson were working on critical gene therapies for various genetic eye diseases and it is crucial that vectors are produced as quickly as possible.

“It is only when we have our own facility right here in Australia that we will be able to speed up how quickly we can translate our current work in the lab to real therapies for patients.’’

CMRI’s Director Professor Roger Reddel thanked the NSW Government for its foresight.

“Investment in this facility will not only advance the impressive gene therapy work being done in our labs but it will significantly benefit the collaborative precision medicine programs across CMRI and our partner institutions across the precinct, including Kids Research, and ultimately, will transform the lives of Australian families.’’