I wonder what the 4 OTHER new programs we tested it on are?
"Following internal corroboration of published results on the MyoAAV platform, Sarepta secures exclusive license for Duchenne muscular dystrophy, plus four additional neuromuscular and cardiac indications
The MyoAAV platform is a potential breakthrough in genetic medicine delivery, with early research showing significantly greater gene expression at lower doses compared to natural serotype capsids."
"--Delivered 25-50 times greater gene expression in multiple skeletal muscles and 10-15 times greater gene expression in cardiac muscle;
---Demonstrated reduced delivery to the liver by 50 percent and showed lower accumulation in the liver;
---Can be used at up to a log lower dose than traditional AAV vectors, due to increased efficiency.
AND IT WORKED!
Or else we wouldn't have bought it. CMT - if it pans out - has many other applications. It alone would be huge. Plus two other programs that remain mysteries, I believe. Seems like the non-human primate testing would have been done on all five drugs (or indications) ... or at least sufficient testing to assure us that they were going to work with this new delivery technology.
Sarepta may have just shown the street - in the last 2 weeks after the FDA nod on DMD - that we ARE going to hit the big time. And it could be coming quiker than anyone suspected now. And so ye'd best place your bets asap, it would seem.