Dinakar Singh

As we dug in more, we then started talking to scientist friends, Columbia, et cetera, and got more intrigued that there was a chance here because there's some really good work had been done.

And in your body,

If you're missing a gene and it's completely gone, replacing it when it's totally gone missing, I mean, true gene therapy is still really, really hard to do.

On the other hand here, you had this backup gene that was defective, but having a backup factory in place that you could try and crank up and fix to make it a bit better is a wholly easier challenge than having to replace something that simply went missing in the first place.

And so that struck me as being pretty interesting.

There was some work being done.

You could create mice that had SMA, which is also a pretty big, powerful factor.

You had an intriguing thing to tackle.

You had some tools that actually were pretty nifty tools in these cell models and mouse models.

So you could test things in them to see whether they had an impact or not.

And this is getting a little nitty gritty, but for a lot of drugs, one of the biggest problems is what they call the therapeutic window, meaning with almost anything in your body,

Too much or too little is pretty bad for you.

And I joke with people that take water as an example.

Water has a very wide therapeutic window.

But by the way, if you drink enough of it, you'll actually get kidney damage and die.

And most other things are much, much narrower.

And so there are many drugs that actually might work, but we just don't know exactly what the precise amount is and where to get it.

And as a result, they end up having toxicity problems or undershoot, etc.,

With SMA, the difference between some of these kids with multiple backups versus not, and someone who is a carrier versus having a disease, or someone like you presumably doesn't have at all, there's such a massive wide range of this protein in the body that it's pretty clear that you almost can't have too much.

There's no real worry about that.