Sana Biotechnology, Inc. Common Stock Citigroup’s Biopharma Back to School Summit 2026

We'll kind of level set and Can add 27 if you want.

Yeah. First of all, I think most people here recognize we'll make forward-looking statements, so feel free to take a look at our 10-Q for risk factors. We've been working hard for a while on developing two different, important, what we think could be important platforms. One is what we call our hypoimmune platform, and that has the goal of hiding allogeneic cells from immune recognition, and it gives us the chance to really scale the use of cell therapy broadly. Our primary asset has been this drug we call SC451, which is in development for people with type 1 diabetes. Our goal is very simple. It's a functional cure. It is one injection of cells that will lead to normal blood sugars or euglycemia, with no immunosuppression for life for these patients. We can go into why that might work as we chat.

But we have made a lot of progress. We've shown that the technology works in a different setting, which was cadaveric-derived islets, which is less scalable, so we really want to develop the real drug. We're very close to being able to figure this out. We've been working hard this year on an IND, and hopefully beginning our clinical trial. Happy to say that so far so good. There are three really important parts of getting an IND ready. One is your clinical package, and that is both trial design and also how you're really going to deliver this medicine to patients. It's a real operational Rubicon to get this type of a therapy to patients. I think we should feel really confident around our ability to move forward there.

Second is a non-clinical or preclinical package, which includes things like GLP toxicology studies and biodistribution and efficacy, and in the case of this, genomics and other things. I think that is now more or less in the rearview mirror. We have preliminary results across the board, and we do not have every final report, but I think people can feel pretty good that that is not going to end up being an impediment to us moving forward. The third is to both lock down a manufacturing process and then to transfer it, along with all your assays and supply chain and things into a GMP manufacturing facility. We have made real progress along that, and we are in the final throes of transferring it in the process or and doing the types of runs you need to kind of validate that this works in that site.

We are not out of the woods yet, but it certainly seems like we are getting very close, and that we will be in a place to start this study soon. Once that happens to go into kind of data flow, right. Because that was part of your answer. I do think that clearing an IND in a complex medicine like this is not a trivial endeavor and probably has some meaningful value. We then will be looking really on a couple of things clinically. One, does what we have seen before, which is this platform seems to work, really translate into this setting. What we mean by that is can we transplant these gene-modified stem cell-derived islets into the muscle of a patient and see them survive and really function over time, right. The most important part of that is evading immune detection.

We should know that within about a month, right. We view that as being super important, because it will tell you that this thing is kind of inevitable, right. Most of the risk is in that. I do not know most of the value is in that. You guys decide where you are going to place value. Once we have done that, then you want to see, okay, do these cells really work as well as you hope they do, and do you have the medicine that you want, right. That could be dose and things like that. There what you want to see is a person have normal sugars, blood sugars, more or less, with no insulin and no immunosuppression. What we have seen in the field is that that generally takes a few months longer to understand, and so we will be watchful for that.

Then the third is how consistent are these results. Is this happening every time, more or less. Is it happening most of the time. Some of the time. That will take a little bit longer, but I think we will learn all of those things through 2027. We also are moving forward into phase I with a process that is good enough for phase I, which we can talk about what that means more in a bit. But it is not really yet at a scale that we are ready for commercial. I think next year we have a real chance, we are making a lot of progress to be able to tell you if that is going to be something that slows us down in moving towards a registration study or if we are ready to go. Those are kind of really important things with that.

Our second platform is an ability to deliver genetic payloads to cells in vivo. I think you recognize, apologies, you can more or less do anything you want to a cell and its genome in a Petri dish. The hard part is doing this inside the body, so we've been focused on delivery for a while. We have a technology we call fusogens, which allows for cell-specific delivery. We're making an in vivo CAR T cell with this. We've made some fundamental assumptions along the way that we think will lead to a better, safer, and more durable effect with this therapy. It's complicated our path forward, which just means we've fallen a little bit behind some people who are ahead of us, but we think we have a chance to really have a best-in-class medicine, and we could again talk about what that means.

But I think in animals, that's kind of been true, right? Now we need to show that in humans. The first drug is a drug we call SG293, and it delivers a genetic payload that in vivo or inside the body makes CD19 targeted CAR T cells. We're initially going to go after this in cancer and safety profile looks good and things, we may go after autoimmune diseases after that. Our goal had been to start that study about now. We're doing this in China through an IIT. Things got a little bit more complicated over the past several months. It's not a matter of if it's going to happen, it's a matter of when. The other day, we kind of let people know this might take us into 2027 to get our data, first half 2027.

It's disappointing to us, but it's not the end of the world. Behind that, we have another BCMA-targeted CAR T-cell that we can develop with the in vivo technology, assuming this works. I think there's a lot of other things behind that. So that's a little bit about the technologies we have and what we'll see over the next, call it 6 to 12 months.

Well, it's a complicated question in a way. So one, whenever you're transferring in technology, as said, you're transferring in supply chain, right? You're transferring in manufacturing, the process itself and making the drug, and then you transfer in the assays, right? All of those things need to be done. Really to kind of put the pin on being done, you really want to kind of validate your assays with product that have been made inside that facility. So give us a bit, that's probably one of the log poles, the last things to get done. But generally, I think we're confident that the assays have been transferred and are in good shape. Do we have what the FDA requires for release and things?

I think we have general alignment with regulators in multiple parts of the world around what we need to do to release this drug. Do we know for sure that they won't ask us to do something else? We don't. I think some of that is always dependent upon their final review and reviewing our clinical protocol and the non-clinical talks and package and all those things. But I think we feel like we're in good shape of knowing what we need to do and being able to do it to make this drug and release it and test it in people.