About me
I'm a molecular geneticist and mid-career researcher with a research focus on the development of antisense oligonucleotides (AOs) and their application in translational medicine. I have 14 years of practical expertise in designing AOs for modulating the splicing and expression of genes in rare genetic and neurodegenerative disorders, including amyotrophic lateral sclerosis (ALS), spinal muscular atrophy (SMA) and Parkinson’s disease (PD). My experience includes the evaluation of AOs in both in vitro and in vivo models, as well as experience working with industry in drug development and FDA regulatory procedures for a rapid pre-clinical translation of AO therapies. Alongside Professor Anthony Akkari, I lead the dually based Motor Neurone Diseases Genetics and Therapeutics group at Murdoch’s Personalised Medicine Centre (formerly Centre for Molecular Medicine and Innovative Therapeutics, CMMIT) and the Perron Institute.
Since 2020, I've built and led a team to establish the first stem cell modelling facility at the Perron Institute to enable neuronal differentiation and modelling for antisense drug screening, with a focus on iPSC-derived motor neuron differentiation.
My AO development has resulted in a number of co-authored publications as well as five international patents and two PCTl patent applications detailing AO therapies for ALS, SMA, asthma and cystic fibrosis. Complementary to developing antisense therapeutics, I've contributed to identifying genetic markers for ALS that are currently being evaluated as tools for clinical trial stratification to improve patient outcomes to treatment. This work has led to the characterization and publication of three structural variant genetic markers that explain ALS risk and survival, with one variant the subject of an international patent.
In the last four years, I've worked alongside Black Swan Pharmaceuticals in the US to develop a therapeutic pipeline for neurodegenerative diseases, including ALS and PD. In 2020, I was appointed as the Director of Therapeutic Development with Black Swan Pharmaceuticals to lead the pre-clinical development of this pipeline. As part of this role, I have been the science lead on Black Swan’s flagship program, developing a SOD1 targeting therapeutic from early phase development, through pre-clinical evaluation, leading a team of researchers and coordinating three national and international collaborative teams. I am a consultant to Sydney based company GenieUs Genomics and am the science lead on a commercial research contract leading a team of researchers to develop personalised medicines for ALS patients.