Dr May Aung-Htut is a molecular geneticist and RNA therapeutics researcher specialising in antisense oligonucleotides for rare genetic disorders and more common conditions, including cardiovascular disease. She is Co-Head of the Molecular Therapy Laboratory at  Perron Institute and Murdoch University’s Personalised Medicine Centre, where she also jointly leads the UltRare Research Group. Trained in biochemistry and molecular genetics at UNSW, her earlier research investigated cellular ageing and the transcriptional regulation of programmed cell death.

Her current program focuses on the design and preclinical evaluation of mutation specific ASO for n-of-1 therapies and ASO for a broader application. This work integrates patient derived cellular models, organoids and splice modulation platforms with rigorous evaluation of oligonucleotide efficacy, chemistry and toxicity.

Her research portfolio spans childhood dementia, congenital muscular dystrophy, Pompe disease, familial hypercholesterolaemia, multiple sclerosis and neurological disorders involving dysregulated microexon splicing. Through this work, she is extending the Molecular Therapy Laboratory’s established translational platform to conditions for which conventional drug development may be commercially or technically unviable.

Dr Aung-Htut has an outstanding record of attracting competitive and philanthropic investment, contributing to more than $20 million in funded initiatives since 2024—including major National Critical Research Infrastructure (MRFF) programs to accelerate antisense therapeutic development and deliver personalised treatments for children with rare genetic disorders. She embeds consumer perspectives throughout therapeutic development, connecting molecular precision with transparent, equitable and person-centred translation. By combining molecular precision with consumer involvement, Dr Aung-Htut is helping move highly personalised RNA therapies from laboratory discovery towards clinical evaluation and patient care.

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