Dinakar Singh

So it's not about going and begging for money.

People, I think, were excited by the idea of joining in to go and figure out whether we could actually go and solve a disease.

One first stop was, look, there were some scientists that had done some work suggesting that some existing drugs could actually modify the disease in mice and things like that.

So we basically created all these mouse models and cell models of different types to test different versions, and each has pros and cons, and set up facilities to essentially give them away for free.

So essentially, we said to everyone around the world, guys,

Send your drug libraries in, your compound libraries in.

Test them for free.

We'll pay for it.

You keep the IP.

We just want you to know if there's something interesting here on stuff.

And again, because no one cared about rare disease, let alone a pediatric one at that time, it took years before any company, even for free, was willing to do that, which is crazy.

Finally, we got Novartis to bite.

And they ended up finding a drug that had failed for tox reasons for anorexia or something back in the 70s that was off the charts good.

And so suddenly like, oh, this is interesting.

To give an example of just how different the world is now versus then.

Early on, I'd use relationships to go drag some of our scientists and some of our advisors, et cetera, to go meet with heads of research at different companies and try and pitch them and say, look, guys, I know this is a small disease, but there's a chance to do something.

It won't cost a whole lot to find out.

You don't have to spend 40% on marketing, all that kind of stuff.

And so one of my slides showed that if you charge $5,000 to $15,000 a year, you could actually end up with a drug worth hundreds of millions of dollars in profit.

And so it wouldn't just be a charity project.