oh wait, that’s what PYC does, but no one gets it @travelbug2One...

oh wait, that’s what PYC does, but no one gets it @travelbug2

One thing we can be confident about is that PYC gets it. If you have powerful forces that yield six-fold success rates you need to play to your strengths. The second criteria on slide 10 for enhancing prospects for clinical success is applying RNA drugs in the context of increasing gene expression.

What does that even mean and do the three programs in PYC's pipeline tick the box! Help is at hand on slide 12 and in particular I recommend the article by Elie Dolgin titled Hope for haploinsufficiency diseases. You will find reference to the Goldilocks paradigm where you need to produce not too little and not too much; the amount of protein produced by the RNA drug has to be just right.

The article quotes a neurologist from NYU Langone Health who comments that an effective therapy in one of the haploinsufficiency disorders could provide a platform to address other haploinsufficiencies. And, yes it would appear from my brief look at the literature that Retinitis Pigmentosa Type 11, Autosomal Dominant Optic Atrophy and Phelan-McDermid Syndrome could all be characterised as haploinsufficiency diseases.