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clock Created with Sketch. 13/08/22
08:51
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Originally posted by hottod:
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I have viewed ProQR as PYC’s closest competitor. Both were small companies focused on antisense therapies for rare eye disease. The key differences have been in the antisense technologies used - PYC uses PPMOs while ProQR has been using naked antisense (ASOs) - and the fact that ProQR was a few years ahead of PYC. ‘PYC’ has successfully completed a competitive evaluation of a drug that includes our delivery technology compared to a ‘naked’ Anti-Sense Oligonucleotide (ASO) that does not. The drug that includes our delivery technology is effective at a dose of less than 1 microgram per eye in a mouse and the drug that does not include our delivery technology is not effective at either 1 microgram or 10 micrograms per eye. The results show that PYC’s delivery platform can make ASOs into potent drugs at doses that are otherwise ineffective – meaning better drugs with fewer side effects. Delivering more drug to the target in a non-toxic manner is the rate-limiting step in the burgeoning field of RNA therapeutics2 – an emerging field in which ASOs are a leading class of drug across a range of diseases. This data provides the central piece of pre-clinical evidence that our delivery platform is capable of achieving outcomes that competitive technologies alone cannot.
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So they’re down, but not out (as a competitor).
(20min delay)