I disagree that I have a great understanding of the science. It’s certainly not my background. There's been a lot of reading required just to get some kind of grasp.
In answer to your questions…..
Yes, the Harvard Mass Eye and Ear/Biogen program would be in direct competition to what PYC is offering. Both are targeting the same patient cohort – those with adRP that is RP11 (~10% of the adRP population ).
A key difference is that Harvard Mass Eye and Ear is tackling the problem through gene therapy vs. the PYC approach of antisense oligonucleotides. If you refer to PYC’s 22 June presentation (Slide 8) you’ll see the different therapeutic approaches listed as well as some of the companies involved.
There is already one gene therapy approved for RP in a different (smaller) subset of patients. That is Spark Therapeutic’s Luxturna, which was approved by the FDA in 2017. This therapy is mentioned in Slide 22 of the June presentation. It costs roughly A$1bn for treatment of both eyes for the one-off treatment. Sound great (other than the price) but the potential side effects are quite scary.
A comparison of ASO therapies vs. gene therapies can be found in this ProQR presentation .
As for funding- that’s a question for the company.
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