(02-24-2020, 05:36 PM)fenders53 Wrote:(02-24-2020, 10:58 AM)Kerim Wrote: Sorry late to this thread -- my speculative play lately has been nibbling on STOK. They are developing a treatment that could really change my daughter's life. As if I don't have enough skin in the game already!
What are they testing Kerim? And best wishes to you and your daughter.
Thank you!
I barely understand it, but at a hugely vague and likely inaccurate level: some genetic-based diseases occur where there is one good copy of the DNA and one mutated copy. In building the body's cells, both copies are read by the RNA, and the things built from the mutated copy don't work properly. (In my daughter's case (Dravet syndrome), it is the sodium ion channels in her nerve cells, which leads to seizures and developmental delays.) STOK has developed a way to replace the mutated bit in the RNA, so that much more of the resulting structures built the right way instead of the "mutated" way. (Here's a way better explanation.) They haven't tried it in humans yet, but it worked like gangbusters in mice, apparently. Phase I human trials are this year. A different company has successfully used a very similar technique in humans for a different condition.
Even if the tech works, I have no idea if this will turn out to be a good investment (there's lots of reasons to think it might not be). But of course I'm not betting big, and I kind of like the idea of partnering up with the company that might really help my kid.