joel12 schreef op 15 juli 2019 17:13:
ok, i posted the thought on the other board by mistake.
in essence if looked in the Proqr presentation
ir.proqr.com/static-files/a04d2d08-6e... page 125
thats the competitive advantage of Proqr. 1 delivery method, which is a chemistry solution, its IP,, which generates various Modes of action based on the desired effect, based on the biological problem to be solved.
So Proqr has 1 programming language call it,, through which it can deliver its RNA tools, be that exon skipping, be that RNA EDITING, be that even GENE SILENCING (gamper from Ions is a gene silencing) and more like mRNA lowering (already entering the space of Moderna)
I dont know of any other company with such a delivery method that can do so many things.
RNAi companies like ABUS, ARWR, DRNA, ALNY are using nanoparticles delivery system to do mostly gene silencing. they dont do rna editing, they dont do exon skipping, etc.
now what to say here?
Proqr delivery is highly versatile and can encompass a world of biological changes, its only bottleneck is to have the right targets ... for that comes in GLPG (for example) which understands biology and validation of targets. then Proqr could come with its toolbox via its elegant delivery and solve the problem as needed.
the competition to is method?
1) gene therapy but its much more difficult at various levels and has more side effects. you cant have 1 delivery method for all targets
2) gene editing, same as gene therapy .. difficult and side effects. limited to which genes you can apply,, definetly not to the BRAIN!
3) gene silencing .. limited in scope to certain genes.. so limited in the scope of what it can do..
4) small molecules --- very difficult to develop, long timeline , costly to manufacture etc.. and for each target need to reinvent the wheel.
bottom line,
Proqr may had found a simple, effective, elegant way to deliver a toolbox of RNA solutions to all the body including for sure the Brain, and such to become the to go company in solving the worlds diseases at speed .. especially by a company that knows biology,, i.e GLPG.