About me
I am an internationally recognised RNA therapeutics scientist, molecular biologist and Emeritus Professor at Murdoch University. I was an integral member of the internationally recognized group that pioneered therapeutic antisense oligomer development, building a 25-year partnership with Professor Steve Wilton. The group demonstrated specific exon skipping in a mouse dystrophinopathy model and was the first to recognize the clinical potential of morpholino oligomers, and to describe a complete panel of oligomers for every dystrophin exon. With Sarepta Therapeutics, we designed the lead morpholino compound (Eteplirsen), the first FDA approved drug specifically for the treatment of Duchenne muscular dystrophy.
The novel compound Eteplirsen (ExonDys51 ®), licensed by Sarepta Therapeutics was granted accelerates approval specifically for the treatment of Duchenne muscular dystrophy (2016) and was the only drug to have altered the natural history of Duchenne muscular dystrophy (DMD). Additional compounds developed by the Wilton/Fletcher research team include Golodirsen (VyonDys53 ®), designed to address another subset of DMD mutations was approved by the US FDA in 2019 and a third compound, Casimersen (VonDys45 ®), was approved in 2021.
Since 2012, I embarked on collaborative functional studies to improve antisense oligomer uptake in the heart and overcome the limitations of these compounds to treat dystrophic cardiomyopathy, a major cause of death in Duchenne muscular dystrophy. The group reported the mechanism that underlies mitochondrial dysfunction in dystrophic heart and showed that treatment with an antisense oligomer, modified to enhance uptake, could partially rescue cardiac function. More recently, she established a collaboration with Dr Fred Chen to develop novel genetic drugs to treat inherited blindness, with the therapy for retinitis pigmentosa being developed in partnership with PYC Therapeutics and Lions Eye Institute. Additional projects to understand mechanisms in motor neuron disease and develop antisense interventions are underway.