Sarepta is already developing the next generation chemistry in DMD which includes the addition of a peptide. The PMO platform is being enhanced for tissue targeting, intracellular delivery, target selectivity and drug potency.
Its right up our alley. As always its a case of good, better or best. Fletcher and Wilton, who have access to the PMO platform will be looking to advance more effective treatments for DMD. Could Phylogica's FPP-based intracellular delivery platform do it best?
One thing for certain, Ed Kaye, CEO for Sarepta will want to find out fast. Approval for eteplirsen is conditional upon proving efficacy in longer term postmarketing studies.
Within this context, SoT's comment that "this work is looking more and more like a licensing opportunity for PYC" could prove to be remarkably prescient.
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