It was November 2013. Sarepta announced that they had entered into an exclusive worldwide licencing agreement with UWA for rights to an extensive patent portfolio in DMD. At the time, Sarepta said that the collaboration could potentially develop best-in-class drugs for the disease. Exon-skipping with a single bound went global. The Sarepta success story had arguably begun in Perth.
Fast forward to June 2019. Phylogica announces breakthrough animal model results in inherited retinal diseases. Indeed, it is early days but this is a story I like - for one its set in Perth. The article identified by Hottod, and received for publication in June 2017, lays out some of the outstanding challenges for the feasibility of antisense oligomer mediated splice intervention to treat genetic eye diseases. One of those outstanding issues is effective delivery; another is the increase in cellular uptake. The extent to which those challenges have been met may become clearer in Seminar Room 612a.
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(20min delay)