Dinakar Singh

Early on, we had gone to them to work on building mouse models and making them free so people could call them up and then send the mice over.

But I'm working with them and setting up actually a rare disease institute.

We're going to have a squad of people to do a bit of what we did in SMA because I hear all these heartbreaking stories and they're the same things that I had 20 years ago.

People that want to make a difference don't just want to go and make random donations that they think will just be like throwing water on sand.

They don't know how.

And like in our case,

We had a lot of resources and a lot of connections and money and could essentially go reinvent or invent the wheel.

But for other people, it's a waste of money.

It's a waste of time, both of which are incredibly scarce when you're a parent with a child who's suffering.

And so our goal is to actually have, they call it the Ghostbusters of rare disease, where if you have a disease, you call us and we'll actually get some scientists to work on it to go and help you put together a plan.

Not caring whether it's with us or not, but essentially help be the brain trust for people to want to go and think about game plans for trying to solve a disease that affects some of the care of them.

Well, some good things and some bad things, I guess, that have happened leading to what I hope will happen next.

on the good side.

You don't have to bash someone over the head to get them to understand that emergencies can be lucrative for them.

I mean, SMA has been, there haven't been that many, but when people have seen the money that's sort of been made on this stuff, people care.

But the challenge remains, which is that

If you're a company working on oncology, even if there isn't a drug tomorrow, you're going to be working on oncology for the next hundred years, and that information can be useful in other things.

The challenge with pharmacies remains that a lot of these are pretty bespoke.

So the difference from 20 years ago is that you don't need to convince someone that it can eventually be very profitable, but you still have to convince them that there's a good chance that they'll be able to go and cure it because that early stage investment is tricky.

There needs to be a much more thoughtful effort working with NIH, FDA, and disease groups to find ways to go and have a much more thoughtful, practical early stage because scientists writing papers and grants is really not a very effective way to get this done.