For those not familiar with her background, PYC’s Chief Scientist, Prof. Sue Fletcher, previously led research on developing antisense therapies for inherited disorders, first at the University of Western Australia and then at Murdoch University.
Together with Prof. Steve Wilton, Sue Fletcher pioneered oligomer induced exon skipping to overcome dystrophin mutations that are the root cause of Duchenne muscular dystrophy (DMD).
Sarepta to date has three approved therapies, all antisense therapies for DMD subtypes - Exondys 51, Veyondys 53 and Amondys 45 - and all designed by Professors Fletcher and Wilson at the University of Western Australia before licensing to Sarepta.
In DMD, mutations in the DMD gene lead to almost no functional dystrophin protein being produced, which in turn leads to muscle damage and progressive weakness, beginning in early childhood. Individuals with DMD typically lose the ability to walk by their early teens and face a significantly shortened life span.
The DMD gene, which is the largest gene in the body contains 79 exons (the exons contain coding for protein production in the body). Amazingly, Profs. Fletcher and Wilson developed antisense oligomers to skip each of those 79 exons, bar the first and the last (see first link).
PYC is truly fortunate to have a scientist of Sue Fletcher’s experience and calibre leading the company’s scientific research.
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