A facility for the development of gene and cell therapies has garnered $25 million in funding from the NSW Government.
The University of Sydney, Children’s Medical Research institute, and the Children’s Hospital at Westmead will apply advanced manufacturing technologies to manufacture clinical-grade vectors for gene therapy in paediatric and adult patients.
A vector carries pieces of DNA to tissues and organs to correct genetic errors. These technologies can cure genetic diseases that cause blindness or liver disease.
According to Ian Alexander, head of the gene therapy research unit at the Children’s Medical Research Institute, the facility will get research out of the lab and into the hands of doctors.
“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,” he said.
Currently, these vectors are imported from overseas, and are costly to transport. In addition, with global supply limited, the application of the technologies in Australia will be benefited by locally manufacturing the vectors, highlighted Leszek Lisowski, Conjoint Senior Lecturer at the University of Sydney.
“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 facility will be part of the Westmead centre of excellence, which operates across a number of medical technology sectors, said University of Sydney vice-chancellor, Michael Spence.
“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,” he said.