And let's assume that PPMOs give better results, than what happens to the skipping 51 trial that is using PMOs? Do they have pre-clinical toxicology work completed for other exons using both chemistries?
Ofelia Marin said:And let's assume that PPMOs give better results, than what happens to the skipping 51 trial that is using PMOs? Do they have pre-clinical toxicology work completed for other exons using both chemistries?
If PPMO ends up being better, then it sounds to me that they would have wasted valuable money/time testing PMO.
seems like a corporate tug of war....you got one company (x) with only PMO, so trying to get there first, then another company (y) that has both PMO/PPMO, trying to find the best treatment possible.
I wonder if (y) was forced to jump in early with their version of PMO since (x) was already ahead of the game instead of waiting until (y's) PPMO was ready?
Again, though, I think the cocktail I mentioned is closer to reach and would possibly be better than us hoping for exon skipping. Exon skipping, if proven to work, to me would be a good back-up plan. I may be wrong, but that's not the vibes i got at conference.
These are details about Prosensa's systemic trial:
The exon skipping trials planned and/or in progress are:
AVI exon 51 intra-muscular UK using PMO (in progress)
AVI exon 51 systemic UK using PMO (planned)
AVI exon 51 systemic USA using PMO (planned)
AVI exon 50 USA systemic USA using PPMO (planned)
Prosensa exon 51 systemic EU using 2'O-methyl (in progress)
And...can we find out how much time is involved in what Steve Wilton referred to as a "few"? Ofelia- will he answer that question for you?
And...if, by chance, a "few" turns out to be a huge amount of time (again, our son just turned 10 and we want to keep him walking) does anyone out there know ways to speed this thing up?