Beam Therapeutics Inc. Common Stock Citigroup’s Biopharma Back to School Summit 2026

Thank you for having us.

Yeah, great. Beam has made a lot of progress. We are moving very quickly now across a broad range of programs, all building off of this very dynamic platform that we have created using base editing, of course, next generation CRISPR 2.0 technology for more precise gene editing. But then building that into a delivery and manufacturing machinery that is now quite mature. So doing both ex vivo cell therapy, but also now in vivo lipid nanoparticle programs, and manufacturing all of that internally. So it is a very significant capability we have built. The programs are now moving very quickly. So risto-cel, our sickle program, is really awaiting its final data cut from a now fully enrolled and dosed BEACON trial, that will become a BLA filing right around the end of the year, which will be our first BLA. So that will be an update we are excited to share.

Then in the in vivo programs on the liver, we have BEAM-302 for alpha-1 antitrypsin deficiency. We just have shared new data there showing really dramatic consistency of that program as we continue to add patients. That's now in a pivotal cohort. We're enrolling patients. We dosed our first patient in July. By the end of the year, we hope to significant progress on enrolling that 50-patient cohort, and continue to move that program forward towards patients. Behind that is BEAM-301, which is a program for glycogen storage disease 1A, the R83C mutation. There we'll have first in human data this year on a couple of dose cohorts for that program, another metabolic type of mutation that we're going to try to correct. Finally, also in vivo PKU, BEAM-304.

We now have an open IND, so we're now opening sites as we speak, and hope to begin screening patients towards the end of this year, and obviously then that'll become a clinical program to follow. So lots going on, lots to talk about, but we're seeing this flywheel continue to gain momentum on both the payload technology and our delivery and manufacturing capabilities.

Yeah, it's a great question. So there's been a lot of enthusiasm, and I'd say ERS is no exception. We're hearing from physicians around the world, certainly our investigators, but more than that this looks like the profile they've been waiting for. This patient community is really overdue for some better therapies. When you ask them what is the ideal outcome, they will say, "Well, we need something that raises our AAT above the protective threshold," which basically means into the teens, because that is the place where there are no patients who have those kinds of levels. We want to raise it by creating M, the normal protein, which we know is functional and will protect their lungs. At the same time, we want to be reducing Z as much as we can.

That is a toxic protein both for the liver, but we have learned a lot more recently about its disruptive effects systemically, causing inflammation and interfering with the protection of normal AAT. You want it to be inducible, right? When you get sick, it needs to go up, and we have shown that. Then, of course, you want it to be durable. These patients have been on chronic therapy their whole lives, taking weekly augmentation. They are very hungry for a one-time cure, and that is what we believe we are offering. Lots of excitement, I think, across the physician and patient community, and we continue to see that operationally in our BEAM-302 trials.

Yes, great question. There are multiple assays available, and I think the important point is to step back and see that they are actually telling the same story across the board. We do change back and forth between them a bit. Turbidimetry is the classic in the field. This is literally taking protein in liquid, shaking it up, and seeing if it disrupts light scattering. So it is a fairly crude sort of old assay, but it is used by the clinicians very frequently. So that is the standard. If you are going to get diagnosed with AATD, you are going to use turbidimetry to do so. But they do not check AAT levels very frequently. It is kind of a one-off. For our first academic conference presentation, which was yesterday, we thought turbidimetry makes sense because it could be familiar to the clinicians, and they would fully understand it. LC-MS is different. That is, of course, mass spec, and we need LC-MS to discriminate between the kinds of AAT, right?

If you want to see how much M you have, how much Z you have, turbidimetry cannot do that. So we need LC-MS at least for that. You have seen some of our LC-MS data. It is reporting that. Our expectation is ultimately even things like total AAT level for the FDA, they will also want LC-MS. So we have been bridging our way over to that assay. I expect you will see more of that from us in the future. Right now we are validating it for pivotal stage. But it gives fairly similar answers. There is definitely for these assays, there is a 10% wiggle as you think about sample to sample. Also AAT levels themselves drift up and down for these patients on that order of magnitude.

I definitely don't see, there's no change in our levels, I think that you should interpret here, between 16, 14.5, it's all basically mid-teens. As we have seen, it's quite durable. Once you see those set points, I think you'll see them sustained.