I agree the catalyst for the delay was the prpf31 patent which links to the RP11 patent resulting in end to end IP protection at IND filing around causality between the two and the process around splicing (intron movements pre-mRNA to exons in post or mature mRNA) to protein formation and the bits in between (formation of the spliceosome… all the way to the splicing reaction) all the way to the degeneration of the retina/vision loss.
This will be a world’s first and validate PYC’s unique approach, even at this pre-IND stage it’s just a very tight value position with the IP now squared away - I can’t help but feel FDA will be impressed with this end to end well thought out approach - if it validates via green light to proceed with trials this will be huge external validation for PYC and can see it rinsing and repeating to scale out its platform lead pipeline
that means clarity on patient population (inclusion criteria for prpf31 means trial end points will be designed for success as biomarkers (we can get ultra specific here with two patents locked in) and success criteria for drug to market is baked into the the trial design at the outset with off-targets risk minimized as recruitment will be specific for who will benefit…
precision medicine doesn’t get more precise than this fellas, very smart strategy both scientifically, corporate/strategic and regulatory friendly - let’s hope they pull it off
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I agree the catalyst for the delay was the prpf31 patent which...
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