Prof. Steve Wilton

Founding Director and Chairman

Steve has a PhD from the University of Adelaide with extensive experience in molecular genetic studies.

Initially involved in finding disease-associated genes and then developing tests to confirm a clinical diagnosis and/or assist in genetic counselling, Steve has pioneered the use of antisense oligonucleotides to modify gene expression to by-pass catastrophic mutations that would otherwise result in Duchenne muscular dystrophy, a severe form of muscle wasting. The US FDA has granted accelerated approval for three “exon-skipping” drugs developed in his laboratory, Eteplirsen, Golodirsen and Casimersen, as treatments for Duchenne muscular dystrophy.

Steve’s research team has become a pipeline of future drug development to treat many inherited and acquired diseases and is currently investigating treatments for more than 50 diseases, several of which are undertaken with national/international collaborators.