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how bright is the light bulb

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    Sifting through the Operational Update, I noticed the following dot point in relation to Progress on FPP Platform Development;

    Phylogica have been able to quantify the approximate concentration of the protein which they have delivered into the cell.

    This piece of data could be quite instrumental in DMD drug development. Although not spelled out, and therefore entirely speculative, the protein referred to may well be dystrophin. Sarepta Therapeutics have been able to demonstrate that eteplirsen increased levels of a key protein called dystrophin that's deficient in DMD patients. The sticking point with the FDA over approval for eteplirsen appeared to centre upon the level of dystrophin expression.

    There is an excellent article in Seeking Alpha titled Sarepta Dystrophin Levels for Duchenne Muscular Dystrophy Drug by BosCaptn published August 4, 2016. There appears to be claim and counterclaim and misinformation but the barney with the FDA over approval appeared to hinge upon two different lab techniques used to measure dystrophin. Sarepta used one technique to show a significant improvement in muscle fibres that were dystrophin positive - was the light bulb 'on' or 'off'. There is another technique called Western Blot which measures the total amount of dystrophin protein present in the muscle fiber - how bright is the light bulb. Without going into detail, it appeared that Sarepta was claiming the importance of switching on the light bulb wheras the FDA was saying the light was not bright enough.

    The question is whether Phylogica have been successful in increasing the brightness of the light! The level of expression of the protein could well become a light bulb moment for Pharma and may explain increased interest in the FPP Platform.
 
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