Anna Greka

And there are other mangled misshapen proteins in the liver, in the lung that we're now studying.

So this becomes what I call a node, like a nodal mechanism that can be targeted for the benefit of many more patients than we had previously thought possible, which has been, I think, the most satisfying part about this story of molecular sleuthing.

Absolutely.

I think, you know, the goal of the Ladders to Cures Accelerator, which is a new initiative that we started at the Broad, but it really encompasses many colleagues across Boston and now increasingly it's becoming sort of a national.

We even have some international collaborations and it's only two years that it's been in existence.

So we're certainly in a growth mode.

But the inspiration was really some of this molecular sleuthing work where I basically thought,

Well, for starters, it cannot be that there's only one molecular node, these T-Med cargo receptors that we discovered.

There's got to be more, right?

And so there's a need, you know, to systematically go and find more nodes because obviously, you know, as anyone who works in rare genetic diseases will tell you,

The problem for all of us is that we do what I call hand-to-hand combat.

Like we start with the disease with one mutation and we try to uncover the mechanism and then try to develop therapies.

And that's wonderful, but of course it's slow, right?

And if we consider the fact that there are, you know, 30 million patients in the United States, in every state, everywhere in the country who suffer from a rare genetic disease, most of them, more than half of them are children.

Then, you know, we can appreciate the magnitude of the problem.

Out of more than 8,000 genes that are involved in rare genetic diseases, we barely have something that looks like a therapy for maybe 500 of them.

So there's a huge mismatch in the unmet need and magnitude of the problem.

So the ladder secures accelerator is here to address this and to do this with the most modern tools available.

And to your point, Eric, to bring patients along, not just as the recipients of whatever we discover, but also as partners in the research enterprise, because it's really important to bring their perspectives.

And of course, their partnerships in things like, you know, developing appropriate biomarkers, for example, for what we do down the road.