Sana Biotechnology, Inc. Common Stock Wells Fargo 21st Annual Healthcare Conference

Oh, thanks for having us.

Sure. As everybody knows, we'll be making forward-looking statements, so please check out our disclosures and risk factors in our most recent 10-Q. We also posted a presentation and an 8-K today. The company was founded really around two technologies, and they're both about to really move into humans and we'll hopefully have a really good idea around how they're working. One is, our goal has always been to really figure out how to scale engineered cells and be able to go after highly prevalent diseases with either cell therapy or gene therapy, depending on how you think about it. The first platform we started the company around was the goal of hiding cells from immune detection. The goal of that, and the most promising program we have there, is a potentially one-time treatment for people with type 1 diabetes.

That's making progress and should be something that we can file with the FDA this year and hopefully start a phase I study. The technology, just to take a step back, type 1 diabetes, about 10 million people in the world have it. If you are 22 years old and you have the choice between breast cancer, HIV, and type 1 diabetes, it turns out that type 1 diabetes will have the shortest expected lifespan. That's with the best therapy, and you can imagine the day-to-day stress of that disease. You may know people with it. It's very difficult to manage. People have challenges with both hypoglycemia or too low blood sugars, and then all the complications long-term of hyperglycemia, or too high. We know a few things about it.

First of all, the disease is one where the immune system attacks the beta cell, the pancreatic beta cells, the only cell in our body that makes insulin. About 100 years ago, it became clear that you could give exogenous insulin, and it went from being an instant death sentence to something people can live for decades with, right? Over the last 25 years, some real progress has been made to understand how we might move towards a more definitive curative therapy, though. About 25 years ago, a group of people, starting in Canada, started transplanting pancreatic islets. Think of an islet as beta cells plus their support structure. They would transplant these islets from a recently deceased cadaver, and people do very well. They can live for decades off insulin with normal blood sugars. It's not a scalable source. It's a very variable source.

It just turns out there aren't that many people for whom lifelong immunosuppression, because this is, again, it's a transplant of cells. It's better than lifelong insulin. Thousands of people have gotten this. Over the last few years, several groups have shown that you can take pluripotent stem cells and mature them into pancreatic islets and transplant them. That's probably a more scalable source. It looks like it's more consistent. But people still remain on lifelong immunosuppression. We've now shown in a single patient, which is all we ever wanted to treat, in the cadaveric islet space, that we can gene modify islets and transplant them into a person with type 1 diabetes. The patient has been able to see these cells function in grafts and persist for now, the last publication was 14 months.

It's been published twice in The New England Journal of Medicine, actually. It's really the first example I think that any of us are aware of, where we have transplanted cells with no immunosuppression. Now all the component parts are there for hopefully a curative therapy, and that's this drug SC451. It's a gene-modified stem cell-derived pancreatic islet. The goal is a one-time intramuscular injection with hopefully a functional cure for the person with this disease. By that we mean normal blood glucoses with no insulin and no immunosuppression, hopefully for decades or life. We'll dose the first patient shortly. Shortly being not defined, but it's coming up on us. The second platform we have is an ability for cell-specific delivery in vivo. The first place we're applying that technology are in vivo CAR T cells.

I have been at this for a while, and we made some very specific choices. We have seen some people get a little bit ahead of us in line, where they have shown some very promising and exciting data in the field. We have worked very hard to ensure cell-specific delivery. We worked very hard to try to minimize the risk of off-target safety events and on-target safety events. Again, that is a program, the lead asset called SG293, which is a CD19 CAR T, so I am sure we will get into a bit more. Our goal has been to get into and start human testing very soon. For clarity, we have been doing this outside the United States, and it is an IIT, an investigator-initiated trial, that we are trying to do in China.

Things have definitely gotten more complicated in China over the course of the last, let us just say 3 to 6 months. It is probably going to take us a bit longer than we hoped to get that done. I do not know exactly what that means. It might be one or two quarters slower to get the human data than we hoped. It will get there, and we have spent a lot of time, again, on this medicine, really trying to create a very clear scientific pathway. We have shown in non-human primates the ability to do cell-specific delivery, meaning we are only getting into T cells, and we see the therapeutic effect we want to. So we are optimistic that this will also have some very exciting data, but you never know till you get into humans and you see what biology gives you.

Yeah. So basically, whenever you are kind of putting together these, beginning of a clinical study, three main work streams, right? You have a clinical work stream of trial protocol, and with this case, because of the novelty and complexity of delivery, that has been a lot of work to ensure we have that. I think we are quite comfortable and confident with where we are on that. The second is your, I call it the non-clinical package, which includes the GLP toxicology study, but includes a lot of other things. That is pretty much complete. All the important studies have been done, and I think we can be pretty confident on preliminary results at least that things are all good. We have to get all the final study reports done. Then we are finishing up the tech transfer into the GMP manufacturing facility. That is still ongoing. We are actually hopefully pretty near completion on that as well.