Sana Biotechnology, Inc. Common Stock Morgan Stanley 24th Annual Global Healthcare Conference

Max, thanks for having us.

Sure. I'm sure everybody here recognizes we'll make forward-looking statements, and like you, I have my own little risk factor. Please read our 10-Q for looking at those risk factors. We started the company around the idea that one of the most important transformations that will occur in medicine over the coming 20 years or so is the ability to engineer cells. We wanted to do this in a way that allowed us to go after diseases that are highly prevalent, with scalable platforms that could access patients broadly. We chose to go after two problems. Excited to say, shortly we should be getting going on studies for them and really understanding their impact on human health.

The first one is, if you transplant someone else's cells into your body, your body will see them as foreign and try to reject them or will reject them. It has been the major limitation for transplant medicine for decades. How we went after the idea of trying to hide allogeneic cells, a transplant from immune recognition. It turns out that the technique we use seems to hide them, at least in some cases, not only from allogeneic, but also autoimmune rejection. Probably the most visible or potentially most important program that we are working on is a one-time functional cure for type 1 diabetes. Type 1 diabetes is actually a pretty simple disease from far away, which is the immune system gets confused and attacks and kills the pancreatic beta cell. The beta cell is the only cell in our body that makes insulin.

Before the advent of endogenous insulin about 103 years ago, patients just starved to death. Their cells couldn't get any sugars over the course of several months. Since then, people have done a lot better, but they still are still not great. Even with the best therapy today, your average type 1 diabetic will live about a decade shorter than someone without it. To put that into perspective, that is about the same for a 20-year-old being diagnosed with type 1 diabetes as being diagnosed with breast cancer or HIV. During that time, it is a very complex life, right? At every meal, every time you exercise, every time you get a little bit sick, you are trying to adjust your insulin. These people deserve something better. If you take a step back and say, okay, the missing cell is the pancreatic beta cell.

I am going to go back and forth between beta cell and islet. Just think of it as an islet is the beta cell plus its support structure. About 25 years ago, a guy named James Shapiro, and others have followed, started isolating pancreatic islets from recently deceased people's pancreases. It turns out it works very effectively. They are transfused in the liver, and people have lived for decades without any insulin. So normal blood glucose is no insulin. The challenge is it is not a very replicable supply source, right? There is a lot of variability. It is not very scalable. People have to be on extraordinary immunosuppression, like an organ transplant. There just are not that many people for whom the risks of immunosuppression are less than the risk of diabetes, right? But there are thousands of people around the world who have gotten this.

Over the last several years, others have begun to show that you can take pluripotent stem cells, either embryonic stem cells or induced pluripotent stem cells, iPSCs, and you can grow them and mature them into pancreatic islets. There seems to be a more replicable outcome. It is kind of working on almost everybody. Two, it seems to be that it is probably more scalable, and I do not think we have answered that question for sure. But people are still on lifelong immunosuppression, and again, so they limit the impact. Over the last several years, what we have shown is we can make some gene modifications. First, we showed in vitro, then in mice, and then in non-human primates, and we showed it in people.

We actually did this in type 1 diabetic, published in the Journal of Medicine, that we can gene modify these islets, transplant them into a person with type 1 diabetes, and they will survive and function. The last update in the New England Journal of Medicine is at 14 months. So no immunosuppression, cells doing really well. So now you have all the component parts together for a one-time curative therapy for type 1 diabetes. What we have is a gene-modified iPS cell-derived islet. So we've made a handful of gene edits. We made a master cell bank, we've grown them, and then now we'll differentiate them into islets. We've been working hard at this. I think we're almost there. Our goal is to get the IND cleared this year and start our phase I study this year.

I'll go through this, but you should know pretty quickly if this works or not. I'm sure you'll ask me questions, but I think it's probably worth a second question before people get bored of me rambling. We have another platform, though, and that is, our goal at that point was to go after another big problem, which is you can do more or less anything you want to a genome in a Petri dish. The hard part has been delivering it into the cells in the body. So we wanted to develop a platform that would allow us to go after the challenge of being able to deliver to any cell in a specific, repeatable, and with any payload way. We seem to have made some real progress on that. So our most advanced program is in vivo CAR T cell.

We've been at this for a while. We have a platform that we believe gives us some very specific advantages we can get into versus others. They've got a little bit ahead of us, so I hope it does play out, that it's been worth the time to make a best-in-class therapy. I'm quite confident if you were a non-human primate, this would be the best-in-class therapy, and whether or not that's true with a human, we'll have to see. But again, our goal is to get that study started soon and start generating data and get that out in the not-too-distant future.

Zero. I said this from the beginning. There's nothing that will get you after about a month from the immune system. So these cells are going to live until they die. When they die, it will be because of something related to these cadaveric islets, most likely. When they live, it doesn't mean that our cells, which are stem cell-derived islets, will live in the long run. If they die, it doesn't mean our cells will die. We got immune evasion, and now we need to put these stem cell-derived islets in and ensure that they last for hopefully decades for people. Unless something really surprising came up, like there's some new twist in the immune system that killed these things, I don't think you learn anything as you watch this for longer. I've kind of been pretty clear on that for a while.

Okay. I don't view any update around us learning that things are still alive as being material.