Finding cures for children's genetic diseases


Major Achievements

1995  Gene Therapy Research Unit formed as a joint initiative of CMRI and The Children’s Hospital at Westmead.
2000 Established clinical grade gene transfer vector production capacity in Australia.
2002 In collaboration with the Children’s Hospital in Paris, conducted first ever gene therapy clinical trial for a genetic disease in Australia.
2009-2011  In collaboration with the Australian pharmaceutical  industry, prepared a gene transfer vector and anti-cancer drug formulation for paediatric clinical trial use.
What’s Next Clinical trials for genetic metabolic liver diseases.