As we head towards seminar room 612a its worth re-watching the TED talk by Steve Wilton Can a drug cure genetic disease? Excerpts from the talk include the observation that "there is potential for this type of therapy to be applied to many many different mutations".
Is eteplirsen just the first drug in this new class of PMO therapeutics? Building a new class of therapeutics can be a life times work. It all began with a blank canvas and an idea.
The story of Stanley Crooke and Ionis Pharmaceuticals is a case in point. The controversy around antisense oligonucleotides has faded, the insurmountable mountains have been climbed and the first drug approved. Spinraza isn't the end of the beginning but proof for a platform. Today, those who were sceptical and most probably some who even scorned the use of antisense, have fallen in love with the platform.
In the case of the labs in Perth, the team that persevered to bring hope to boys with Duchenne will undoubtedly also be looking ahead. In the rear vision window is eteplirsen, up ahead any number of 7000 genetic diseases. The work that was experimental has matured into a platform that is scalable. The rise of the genome and the age of personalised medicine is at hand.
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