Molecular Therapies

Research Focus

The Molecular Therapies Research Group is led by Professor Steve Wilton AO and Dr May Aung-Htut and is located at the Centre for Molecular Medicine and Therapeutics (CMMIT) at Murdoch University (a joint venture with the Perron Institute).  In September 2016, the USA Food and Drug Administration (FDA) gave accelerated approval to a new treatment for Duchenne created by Professors Steve Wilton and Sue Fletcher and the Molecular Therapies team. Eteplirsen (Exondys 51) is the first dystrophin restoring drug of its type ever approved by the FDA. In December 2019, the FDA granted accelerated approval for golodirsen (Vyondys 53), targeting another subset of Duchenne. In February 2021, the third FDA accelerated approval was granted for casimersen (Amondys 45). The three drugs developed by Professors Wilton and Fletcher can treat almost thirty per cent of patients diagnosed with Duchenne.

The causes of Duchenne muscular dystrophy are genetic. Genetic mutations interfere with the production of muscle proteins needed to build and maintain healthy muscles. Similar disruptions are found in spinal muscular atrophy due to a problem with the SMN1 gene not making a sufficient amount of protein required for motor neurones to work properly. This leads to the breakdown of these motor neurones, so they do not send signals to the muscles.

The focus of our Molecular Therapies Research Group is the use of small genetic ‘patches’ called antisense oligonucleotides (AOs) to mask part of a genetic message associated with a particular inherited disease. In the case of Duchenne muscular dystrophy (DMD), the defective genetic message is associated with the gene for the protein dystrophin, which plays a pivotal role in maintaining muscle structure and integrity.