CAMP4 Therapeutics Corporation Common Stock 12th Annual Cantor Fitzgerald Global Healthcare Conference

Yeah, absolutely. Thanks for inviting us as always, Steve. Appreciate being here. Maybe I will do a little bit of storytelling, but I will- Quickly.

A little bit of storytelling, but I'll start with some background. SYNGAP1 is a genetically defined rare disease. It is caused by what's called haploinsufficiency, which means you have one remaining healthy gene, and essentially you're missing 50% of what you'd otherwise need to be healthy. The genetics are very elegant. What we know about this disease is that SYNGAP is a very important protein that regulates the AMPA receptors. Meaning, if you do not have enough SYNGAP, you have too many receptors in your synapses, and you get too much synaptic firing, which leads to many different downstream sequelae, learning disabilities, cognition problems, seizures, anger. Anything that essentially you think could affect these children and adults, because there's no early death here, is plagued by this disease, all caused by this missing protein.

The storytelling part of this is what's really interesting, having spent 25 years in rare diseases, is every 3 to 5 years, you learn about a new rare disease that causes you to say, "I've never heard of that," and then it becomes a very important rare disease. Examples would be SMA, Dravet syndrome, TTR, on, and on. Last year, when we began talking about SYNGAP, I think everybody we spoke with started with, "What's SYNGAP?" What I can tell you now is, we never get that question anymore, which I think is important and speaks to the importance of this disease. A lot of credit given to the patient society and the patient organization, who's really done a wonderful job of getting the word out there.

The other thing I can tell you is these patients keep popping up everywhere, and in the public setting as well, and more recently, Becky Quick on CNBC said, "I have a daughter with SYNGAP," and others. We keep hearing about this. I think this is going to be one of those rare diseases that's not so rare. It's a really important approach we're taking because there's nothing out there. There's nothing but unmet need. Nobody's ever developed anything. We're the first ones to do that, which I think creates an opportunity we'll talk more about. But to start with, we got to do something for these patients.

Yep. So one quick update, we are now approved to start in the U.K. as well.

Okay. Congratulations. We'll send a press release for that tomorrow, and we're working on the rest of Europe.