Good afternoon. Thanks everyone for joining us. I'm Salveen Richter, a biotechnology analyst at Goldman Sachs. We're really pleased to have the Bluebird team with us. We have Andrew Obenshain, CEO. We have Tom Klima, Chief Commercial and Operating Officer. To start here, it's been a really busy time for you guys, given the launch of, you know, two drugs and two different indications here, with the potential for a 3rd in sickle cell to come. Just help us understand, maybe starting with ZYNTEGLO. We can go through these programs. Approved in August of last year. The first patient was infused in April. Can you just walk us through your launch strategy and how it's maybe, if it has changed at all, during the period and the process and, you know, where bottlenecks have been or, and where they've maybe been alleviated and kind of where you stand today with that? Thank you, Salvene. I think, first of all, this is just a actually wonderful learning opportunity, to be able to launch a gene therapy, actually two at the same time, in the U.S., and actually really prepare for the launch of the large opportunity of lovo-cel at the end of this year. There certainly have been a lot of learnings, but maybe I can just back up and just explain just how gene therapy is different in terms of the dynamics, of a launch. There's two elements that we really talk about quite a bit and we guide to, which are super important. Number one is the number of treatment centers that we set up, and I'm going to lump in the SKYSONA and ZYNTEGLO launches together here. We initially set up five treatment centers, five centers where people could be treated in our initial launch. From that initial five, we've generated 10 first collections. Now, as of May 9th, our last announcement, we're up to 13 centers, and we're scaling to 40. What that means is that from five, we generated 10. Those, nine of those 10 were in this year, right? You know, a collection is not revenue, but it's the potential for revenue. That's those 9 collections represent a potential revenue of $26 million. That's a lot of value creation for a launch of two very, you know, small orphan gene therapies. We're going to scale those. Just from five centers, we're up to 13, and we're going to 40. By the time we get to this lovo-cel launch, we'll have 40 centers ready to go, and that's the advantage of having an 18-month head start, right? That's the advantage, to get that set up so that we're ready for that lovo-cel launch. I think what we've learned through the launches is that the demand is there. There are plenty of patients that want the therapy as we thought. We've also learned that the payers are willing to pay for it. We've had zero ultimate denials, and I think both of those were open questions at the beginning of this launch. Now where we are, is we're really just into scaling the launch, moving forward, getting more collections, getting up to those 40 treatment centers to be ready for lovo-cel. Okay, I have a lot of questions with regard to that. You have, I think, for ZYNTEGLO, an upfront payment paired with an outcomes-based agreement. Do you think that's really kind of what's gotten these payers, you know, over the line to be comfortable with this payment model around your assets? Yeah. Thanks, Salveen. Just to build on what Andrew was saying, you know, our strategy was founded on three key things. Number one was our engagement in the patient community, and everything we did was to ensure that patients received therapy or were going to receive therapy in the future. Our QTC network was designed to bring our QTCs to where patients live, so they could ultimately not have to travel to get treatment. Our access and reimbursement strategy was really built on speed of access and quality of access. By speed, I mean shortly after approval, and by quality, I mean coverage to our label and clinical trial criteria. It's really exciting now to see both of those things kind of check the box and happen and on to zero ultimate denials. The outcomes-based agreement was one mechanism to help achieve that. It wasn't the full story. We've kind of led the way when it comes to talking about value, demonstrating value, and then tying outcomes based on the value in chronic conditions with a one-time, potentially curative therapy. We've seen great progress with patient demand, as Andrew called out, great progress with our QTCs and really a great partnership with payers and progress on that front as well. You talked about the QTCs here. When you look at these collective QTCs, how many patients do they account for, or% of your kind of target patients in the United States for each of these indications? Yeah, probably the most clear is ZYNTEGLO and SKYSONA. We'll start with that. With SKYSONA, we've said publicly that we expect to treat between five and 10 patients this year and on an annual basis. That's really because, number one, it's a rare disease, where the patients are pretty well established and easy to define, but it's also a rapidly progressing disease, where when they start to progress, unfortunately, if they don't get treatment, they ultimately die. That's a very much easier thing to predict. With beta thalassemia, patients are also very eager, but they can treat or go through treatment based on their life schedules. It's a little bit more difficult to predict. When you look at the first five centers that we brought on, we had said that we believed about 50 patients were in those first five centers, and we're building to a network of between 40 and 50, and that will unlock, we believe, about 850 patients or access to 850 patients, which is the number of patients in the United States that we believe are eligible for gene therapy. That network also puts us within close proximity of about 95% of the sickle cell population. When we get to between 40 and 50 QTCs, that will represent proximity to about 95% of the sickle cell population that live within 200 miles or less of the QTCs. These would be the severe patients? These are the roughly 20,000 patients who have more severe sickle cell disease. Got it. Got it. You, you commented earlier that the demand is there, right? And there still seems to be some debate from the investor community about, I would say, beta-thalassemia and sickle cell, and whether these patients are going to take these preconditioning regimens and, you know, then choose to get on drug and bank their eggs in the interim if they're women. How, you know, just help us understand, like, are there, like, when you're hearing patient feedback, are there any bottlenecks here from the demand side for the severe patients? Yeah, we're extremely pleased with what we're seeing on the launch front, both from in terms of patient demand, but also, keep in mind that right after a launch, we had roughly 30 QTCs calling Bluebird and wanting to be QTCs. They're doing that because they have patients that are suffering from beta-thalassemia that want to be treated. You know, we don't think of it as bottlenecks. We think of it as a time-bound build and a linear build, where when we had you know, our first five QTCs, that unlocked access to X number of patients, and then we keep adding on QTCs, and really, the bottleneck is just time-bound. Some, as I mentioned, some patients are wanting to be treated as soon as they possibly can. Others will wait for a life event, like a wedding or a graduation or something like that, and they're scheduling treatment a little bit further out. On the first quarter EPS call, you announced that you had 6 patient starts. Can you just give us an update on how many additional, I don't know if you can, additional patient starts have happened since then? Where you stand with, you know, these patients kind of progressing through the, I guess, you know, initial period to when they actually get dose narrowing? As of May ninth, we'd had treated a total of, or sorry, treated, collected a total of 7 beta-thalassemia patients, including 1 in Q4 last year. We collected 3 SKYSONA patients. They are... We did announce that we had infused our first SKYSONA patient in Q1 and our first ZYNTEGLO patient at the very beginning of Q2. We'll give more updates at the Q2 earnings. Got it. Are these patients mostly adult patients at this point, or are they pediatric patients? Any early launch trends that you're seeing as to where the adopters are? It's super interesting because we've seen a, just a wide variety of interest. We feel it's a little too early to say that there's a trend, but, again, I think we're excited with the influx of requests and interest being from a wide range of patients across the United States, including some international patients. It's, it's a, I think it's an exciting time for patients to have the opportunity to pursue a one-time cure, curative therapy, but it's a little too early to say, kind of a trend of the types of patients we're seeing come in. The international patients, I mean, how does the payment situation work for the international payment? Yeah, I mean, we're not going to get into details on how the payment situation works there. I think, you know, it's a little, again, too early to tell, but in some cases, you know, there are mechanisms for that, and depending on where they come from, there are different, you know, avenues. Bluebird is not involved in that. Right. It's the QTC that does it. Got it. Got it. The QTC. Okay that actually manages that, and then, and has the relationships. A lot of these transplant centers have existing relationships with other countries, where patients will be sent in, and they'll manage the reimbursement, and with the, with the other country. Yeah. Let's switch to sickle cell. You filed the BLA here. We're looking to, you know, a PDUFA by year-end, potentially. Could you just walk us through any factors that would differentiate your launch in sickle cell from beta-thal? From beta-thal. Go ahead, Tom. You know, we believe that we have a clear head start, you know, when we look at the market in general, because we are establishing our acute qualified treatment center network now. That's 40-50 qualified treatment centers that should be up and running by the time we hopefully get an approval for sickle cell. The softer lens behind that is we're building relationships. We're learning through the launch of ZYNTEGLO. We will apply those learnings to the launch of lovo-cel. We're becoming more and more of a trusted partner day by day, and I think it's that partnership and that transparency that Bluebird has continued to demonstrate that will really set us apart going forward. In terms of dynamics of the launch, again, I think it's going to be a linear build. With sickle cell disease, the way to think about it is it's still going to be a linear build, but it's going to be starting from a much higher base. When you look at starting with between one and five QTCs versus starting with 40 to 50 QTCs, you can imagine that it's much more amplified when you think about not only a higher patient volume, but also an established qualified treatment center network. When you talk about the linear build that's playing out in beta-thal and that would apply to sickle cell, is that? You know, one would assume it's reimbursement. Like, as payers get used to the dynamics for these QTCs and the paperwork required, that there could be an acceleration in the context of demand. Is that not gonna be the case? Is it because of just the infusion centers? Like, what is it that you know, causes you to think it's gonna continue to be linear? The way to think about it is that it's not an acute disease, neither beta-thalassemia or sickle cell. They're both devastating, but it's not acute. Qualified treatment centers are trying to figure out how to plan, mostly from a financial perspective, on being able to treat a good number of patients with a therapy where, you know, they believe they're going to get reimbursed, but in some cases, it takes a little period of time. We hear a lot that the price might be a barrier. That has not been a barrier. We continue to lead the way in value demonstration or that it's going to be reimbursement. It's really kind of the underlying financial picture of the QTC, where if you can imagine, if they started 20 patients at the same time... $2.8 million in the case of ZYNTEGLO, that puts a huge strain on their balance sheet. QTCs are trying to figure out how to appropriately stagger patients to treat them based on, you know, their, the patient's needs, but also the QTC needs. Actually, that lines up well with the fact that patients are trying to schedule this into their life, right? They all have different life events at different times, right? They're not all coming in at the same time. The combination of the hospital wanting to spread them out, and the patients naturally just having a different cadence in which they want it, just leads to a linear build. On pricing, would we anticipate the price to stay intact as you go to lovo-cel, or is there going to be a change in pricing here? Our philosophy is really to base our price on value and the value that a one-time potentially curative therapy brings in a devastating disease. We did, you know, obviously, a complete assessment for both ZYNTEGLO and SKYSONA, where we feel completely confident about the value those therapies bring. We are doing the same right now with lovo-cel, and at the same time, we're engaged with payers to understand what, you know, an innovative payment plan might look with payers for a completely different disease state with a much larger population and a different clinical endpoint. You know, we're taking a value-driven approach, and we will announce the price of lovo-cel, hopefully, upon approval. Got it. It'll be a similar reimbursement paradigm that you've, like, the outcomes-based agreements that you've had, you're going to apply them here as well? It will be a different approach when you think about what that agreement might look like. We believe that outcomes-based agreements in gene therapy are kind of an ante to get into the game, so to speak. Again, we really want to ensure rapid access and then quality access. Everything we do will be based on achieving that so that patients can ultimately be treated. Let's fast-forward towards end of year, and you've got Vertex, CRISPR, coming in with exa-cel. Do you get a sense from physicians that they view the products at all differently or not? Do you think the head start that you've had with these QTCs and the relationship that you've been building helps you in that in that standpoint? I guess what I'm trying to understand is, there's obviously all these patients, how do you think two players play out with the infrastructure that's out there and the payer models? You've kind of done a lot of the legwork here. Tom's talking a lot today. That's why, you know, we're a commercial company. We have a Chief Commercial Officer up here. Go ahead, Tom. Fundamentally, we believe that these patients have gone, both in beta-thalassemia and sickle cell, vastly underserved for a long period of time. Fundamentally, we believe that more therapeutic options for these patients is a good thing for everyone. If you just look at the sickle cell market, there's about 100,000 patients in the United States with sickle cell disease, about 20,000 with more severe sickle cell disease. One could argue that we could both treat patients and both be successful. Having said that, we believe strongly that our head start with qualified treatment centers is going to be a big differentiator. We also believe that the foundation we're laying with payers and our value framework is going to be a big differentiator. This head start that we have, where we've been treating patients since the end of last year, that will continue to be a huge advantage for us, and the relationships there. When it comes to the clinical profiles, at least what we're hearing from treating physicians and patients, is that they see them as basically completely identical. They're really looking at things like, Which companies do we prefer to work with? You know, who can deliver product consistently and reliably, and who's more transparent, and who includes us in some of these decisions? We've been working our QTC strategy, but also keep in mind, we've been engaged in the patient community in sickle cell disease for over a decade now. When it comes down to a patient saying, you know, which therapy they prefer, being engaged and being transparent with the patient community becomes a big thing as well. I'll layer on here. There is a huge disconnect between, I think, the hesitancy of the investor community in looking at the opportunity and when you talk to KOLs. It's two different worlds. When you talk to physicians, they view these therapies, and first of all, we have 32 months of follow-up on 35 patients, right? We just saw the report on 17 patients. Overall, the clinicians are excited to have two options, and they don't differentiate that much between them. There's nuances between them. If you go to the investor community, there seems to be this perception of a wide difference, which simply doesn't exist. What's going to differentiate us is our head start right now. It's being on the market 18 months in advance and actually preparing the market. I do think I'll just, To say, and I think of the valuation gap between us and the, you know, our competitors, is a head-scratcher based on what we hear from the markets. Sure. You talked about manufacturing. Is there anything with regard to capacity or vector supply that could be a gaining factor to helping these patients? Vector supply, I think we have plenty of vector, then capacity, the drug processing, the capacity is something that we will scale up as, you know, with demand, right? We have the ability to scale that up. We are very focused on the learning from this ZYNTEGLO launch. One of the things, opportunities that gives us is to make sure that we can maximize and take all the learnings we can to make sure that we have a quality release every time, so that we don't have to remanufacture a patient. Also to make sure that we understand how to scale, how to increase the throughput through a clean room, increase clean rooms, et cetera. I do think an ability to have a slot available and to release on time and to make sure that you successfully manufacture and don't have to remanufacture, are going to be competitive differentiation. We're practicing that now with ZYNTEGLO. To reach a broader sickle cell population, it seems like you would need a gentler preconditioning regimen, and I know you've been working on these efforts for a while now. Just remind us what they are and where they stand at this point? I think we absolutely see the need for a general conditioning regimen. I do think it will open up even more market opportunity if that exists. The progress there in that field has been disappointing. There have been a number of failures. There hasn't been a lot of compelling data there yet. We are not currently working on anything internally, and that is because we believe that there are a number of innovators out there that are gonna partner with everybody, right? They're gonna develop this and want non-exclusive partnerships. We're monitoring a lot. We do have discussions with different parties quite frequently. It's something we think is very important, but we don't think this area is mature yet. We're waiting for it to mature. I guess, how do you think about the future dynamics, right? If there were, I guess you're on top of it as a, you know, Vertex on the preconditioning side. How much do you worry about an in vivo program? Well, I mean, certainly, that is the right thing for the sickle cell disease and for beta-thalassemia, and for gene therapy in general. I think an in vivo solution is really would be ideal, not only for the U.S., but ex-U.S. That team is farther off. Yeah Than reduced toxicity conditioning. I think we're talking at least a decade, if not more. Again, that's something that we monitor. Again, that's something that's really far off at this point. Is that anything you can be involved in, as you think of the future portfolio? As we scale, absolutely. I think one of the, one of the things that we have as Bluebird, is that we're one of the few companies out there that can do both the manufacturing and the commercial side. On the vector, on the cell processing, and then have this presence in transplant. Our future is to go out and look at for other opportunities to bring other modalities on to that, to that platform, right? Other people need these expertise that we have. You know, ultimately, we would like to see is maybe that starts with ex vivo. Ultimately, of course, in vivo would be where I see the whole gene therapy industry going. It's already there in some cases with AAV, of course, but I see this moving to more in vivo opportunities for sickle cell. Do you think in any way, you know, the, the company being new to commercialization or maybe not having had these relationships with these patient populations or kind of the cash aspects? have in any way constrained you for this launch? You think it doesn't matter, this is a rare disease launch, you can. You know, you've focused on a territory, you've got the payer side done, and now it's all about unlocking these QTCs in the patients. I'll go first. Yep. We've been through a crucible, right? It has focused us. We have gotten rid of a lot of distractions. We have over 350 people at Bluebird focused on only this, all right? We have IT people that are customer-facing. We have legal people that are customer-facing. Our entire company is living this right now. It is an incredible amount of focus, that gives us the ability to compete in a rare disease market, it is a rare disease market. It would be a different case if this was a primary care market or something else, we would not have the scale. With, with our current size, which is not insignificant, you know, we guided to a cash burn of $270 million-$300 million this year. With 350 people, that's a lot of resources to put against a rare disease launch. We do feel that we do have the resources. Yeah. One thing to add to that is I think we arguably hired one of the most talented and experienced launch teams when it comes to selling gene therapy. We've hired a footprint that has done this and been there before. To Andrew's point, this is a highly specialized process. This isn't something you can plug into a bag, so to speak, and, you know, hope that it kind of fits with three other products. This is different, and it takes a unique skill set. Pivoting to the growth strategy versus R&D. Recognize the focus is on basically launches in three indications, which is a lot. When do you start thinking about, you know, the future growth via the pipeline? Like, how are you thinking about that aspect? I think you've had some programs sitting there. Yeah. We have nothing internally right now besides those three, and we have to earn our way there. Okay? We have to earn our way through these launches and prove that we can do it. Once we do that, we earn the right to go out and to start looking at who else needs these capabilities, who could we partner with? There are a lot of both private and early-stage public companies that are facing a very large spend to develop a gene therapy infrastructure or a commercial presence, and could really benefit from a partner like Bluebird. If you look at who has the capabilities to do what we do right now, it's not very many companies in the U.S. It's us, it's Johnson & Johnson, it's Gilead, it's BMS, and soon to be Vertex. It's not a very large group. Fast-forward 10 years, I think every single pharmaceutical company is going to have a gene therapy arm. All right? We are one of the few that has that capability right now, one of the few that people can actually go to. If we're gonna rebuild our pipeline, it's really gonna be through external innovation. Yeah. You talked about partnerships. Any interest still in partnering the ex-U.S. opportunity for ZYNTEGLO? Absolutely. The door's open, by the way, if anyone wants to come in. I think a couple of things need to happen. I think we are proving right now the U.S. model, and that's there. I also think how the beta-thalassemia, sickle cell, and hemophilia products deal with reimbursement in Europe is really important. We have to see whether the European system is going to accept some value-based prices for those therapies that make those businesses sustainable. If that happens, I do think that really increases the chances that our products will see the European soil again. That has to happen first, and that won't be us going back. That won't be Bluebird, that would certainly be through a partnership. Yeah. That is amazing to see the number of gene therapy launches that are gonna play out or editing, like, in a short time. Yeah, it really, it really is. I think this could be very interesting to see what happens. Yeah. True. With that, I'm gonna open it up to the audience for any questions. One there. Thanks. Do you anticipate adding additional QTCs in 2024 to supplement the sickle cell population? If so, what would be the incremental add-on there? It's a good question. We had guided that we would get to between 40 and 50 QTCs this year. That was really designed with the idea of getting QTCs within, you know, roughly 200 miles of 95% of the sickle cell population. However, that's not to say we won't add future QTCs. When we look at adding a QTC, there's very little cost on the Bluebird side for setting up a QTC, and then if they just treat one patient in a short amount of time, then it becomes worth it. We would see adding more QTCs, but we're not commenting right now on what that holistic footprint could look like. It's not a giant, massive footprint, but it's probably bigger than 40 to 50. Thank you. Just curious if you could talk about your ability and progress to get on Medicaid state formularies for sickle cell, given the preponderance of those patients. Good minority, if not majority, in certain states are Medicaid. Yeah, sure. It's a good question. Just to kind of back up a little bit, the population, we believe, for ZYNTEGLO is about 70%-75% commercial. Again, we've seen no ultimate denials. We are working closely with state Medicaid agencies, going back many years now, at what an outcomes-based agreement that might be digestible for a Medicaid system could look like. Obviously, we're working to educate them on what a gene therapy could bring to their patient population, how many patients they might have within their system. We're seeing great progress, and we'll continue to kind of map out what an innovative payment plan might look like that could be digestible for a state Medicaid agency. Thank you for that. Just as a follow-on, do you have a timeline in mind as to when, you know, the big states, Texas, California, New York, et cetera, might be under such a plan? It's an interesting question. I'm not gonna say which states, we've already seen some states come out and publish, you know, gene therapy or expensive or high-cost therapy policies. We believe that in some cases, that could translate into sickle cell disease and benefit sickle cell. I don't really want to give a timeline for each state specifically, certainly we have the high population for sickle cell states on our maps and are working closely with those states. Yep. I guess to end here, Andrew and Tom, is there anything you want to highlight about this launch that we didn't talk about, with regard to kind of the trajectory and outlook? I think I'll just repeat that the, you know, this is a very different dynamic. We spend a lot of time talking about QTCs and talking about first collections, because those are really the value-creating moments in those launches. Revenue will follow. It inevitably follows from those collections. I just encourage everyone who's following Bluebird, right, to do a little math in the back of their head about what each collection means, and to really track those with us. We're very excited about this. You know, we've been working on this for 12 years, and now to actually be able to be treating patients, to be talking commercially about 2 and very soon three of these therapies is just fantastic. I'll just add to that. You know, the partnership with our qualified treatment centers in some cases, goes back a long time into the clinical setting. They're excited to be our partners, bringing these therapies to their patients, or us helping them bring their therapies to their patients. We're learning a lot through the ZYNTEGLO process with them on how we can improve the process for patients with ZYNTEGLO, but also how we can improve it for sickle cell. This partnership and the early start that we have is gonna be a benefit for Bluebird. Yeah. Great. Well, with that, thank you so much. Thanks, Salveen. We appreciate it. Thanks, Salveen.