Steve Wilton AO FAHMS BSc PhD

I hold the Foundation Chair of Molecular Therapy at Murdoch University and have dual roles of Deputy Director of Murdoch's Personalised Medicine Centre (formerly Centre for Molecular Medicine and Innovative Therapeutics) and Director of the Perron Institute. I jointly pioneered the use of ASOs to overcome some of the most common dystrophin gene defects causing Duchenne muscular dystrophy. My group designed the first morpholino oligomer targeting exon 51 in the dystrophin gene, eteplirsen (Exondys 51) and working in partnership with the US company, Sarepta Therapeutics showed that it restores dystrophin expression and slows disease progression in Duchenne patients. This compound was given accelerated approval by the US Food and Drug Administration (FDA) in 2016 and is now in use in patients. A second compound, Golodirsen (Vyondys 53) targeting exon 53 using a sequence was granted FDA approval in late 2019 and yet a further drug, Casimersen (Amondys45) was granted FDA approval in February 2021. The three drugs provide clinicians with the tools to treat over a quarter of Duchenne cases. We have been extending our research on the therapeutic applications of antisense oligomers to other inherited and acquired conditions, including other muscular dystrophies, cystic fibrosis, motor neurone disease, multiple sclerosis, Alzheimer’s, Pompe’s disease, congenital muscular dystrophy, Huntington’s, asthma and neuroinflammatory conditions.