Sneak Peek of the 2026 Nano-rare Patient Colloquium

Yeah, well, we're well over 60 patients that are being treated now.

I think 65 or something like that.

And we certainly hope to be nearing 100 here before long.

So it's come a long way.

That constitutes almost, what, more than 95% of all the nanorare patients who've been treated with an ASO have been done at Enlarm.

And we're proud of that.

We've grown much more rapidly than I expected because we needed to respond to the demand.

And the research team now is, I think, probably somewhere in the range of 15 people or something like that in research, not research and development.

And that team... is involved in ASO discovery.

And of course, we are fully automated, as you would expect.

We have an AI-informed algorithm that has steadily improved, that enables design of ASOs more effectively and reduces the cost and time in screening that's required, and that continues to advance.

Some part of that team pretty much on a daily basis is working on thinking about how to integrate the information we have into our algorithm that excludes certain sequences and certain combinations of characteristics to save the time as we make ASOs and so on.

So that goes on pretty much every day.

Then we have a group of people who will be then ordering plates from IDT, our plate ASO manufacturer that we put into business long ago at Ionis.

And then we order those plates depending on how many patients that we're trying to treat.

But each patient is a plate of ASOs to evaluate.

And that's directed to managing the problem they have, whatever genetic information that we have about their genotype.

And those assays begin with a single concentration of ASO.

And then once we identify, let's say we screen maybe 400 or 500 sites in an RNA target, and we identify out of there maybe 40 or 50 that look promising, then we make more of those 40 or 50.

And then we do a full dose response studies on each of those.

And that's all done in 300 or 480 will plates.

Um, and it's all automated.

And so there's a lot of work getting the drug in to the plate and getting the plate into the robot and then then the robot does the work and then we collect the data.

And then out of that there we'll identify, you know, 20, 30 good looking ASOs typically maybe more.

Then we do a bunch of other studies to assure that we're getting rid of any ASOs that are unnecessarily inappropriately pro-inflammatory.