Children’s Medical Research Institute welcomes vector investment

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The Children’s Medical Research Institute has welcomed a $25 million commitment by the NSW Government for a new advanced viral vector manufacturing facility at Westmead.

The facility will produce specialised gene therapy tools at clinical grade. The aim is to speed up research and translate it into cures for serious genetic diseases affecting children.

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.

One facility can produce vector to treat a range of illnesses but it can be extremely costly to get it to Australia and the waiting list is long.

According to 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, the manufacturing facility would be a boost to the 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. 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.”