Thank you for standing by, and welcome to the bluebird bio First Quarter 2024 Results Call. All lines have been placed into a listen-only mode. After the speaker's remarks, there will be a question-and-answer session. If you would like to ask a question during this time, simply press star followed by the number one on your telephone keypad. If you would like to withdraw your question, press the star one again. Finally, you're reminded that this conference is being recorded. I would like to turn the call over to Courtney O'Leary from Investor Relations. Please go ahead. Good morning, everyone, and thank you for joining our first quarter 2024 results call today. My name is Courtney O'Leary, Director of Investor Relations at bluebird bio. Before I begin, let me review our safe harbor statement. Today's discussion contains statements that are forward-looking under the Private Securities Litigation Reform Act of 1995, including expectations regarding our future financial results and financial position, in addition to statements of the company's plans, expectations, or intentions regarding regulatory progress, commercialization plans, and business operations. Such statements are based on current expectations and assumptions that are subject to risks and uncertainties and involve a number of risk factors that could cause actual results to differ materially from projected results. A description of these risks is contained in our filings with the SEC, which are available on the Investor Relations section of our website, www.bluebirdbio.com. On today's call, Andrew Obenshain, bluebird bio's CEO, will provide opening remarks. Then Tom Klima, Chief Commercial and Operating Officer, will discuss progress on the commercial launches of LYFGENIA, ZYNTEGLO, and SKYSONA. And finally, Chris Krawtschuk, Chief Financial Officer, will provide a financial update before opening the call up for Q&A. With that, I will turn it over to Andrew. Thanks, Courtney, and thank you, everyone, for joining the call this morning as we provide an update on our first quarter 2024 results and recent highlights. I'm thrilled to be here today on the heels of our first LYFGENIA commercial cell collection, which we announced earlier this week. Nearly a decade after we began clinical development for LYFGENIA for sickle cell disease, seeing a patient initiate commercial treatment for our gene therapy is a monumental milestone, both for Bluebird, for the researchers and the clinicians who worked tirelessly on this program, and also for the sickle cell community, who have been our partners every step of the way. Now, with our first LYFGENIA patient start completed, and with a strong, established commercial foundation in place, Bluebird is poised for accelerated growth throughout the rest of 2024. We look forward to sharing more updates as momentum builds. I will now hand the call over to Tom to discuss the progress in our commercial launches in greater detail. Thanks, Andrew, and good morning, everyone. This week we achieved yet another amazing milestone in our gene therapy journey at bluebird bio with our first commercial patient start for LYFGENIA. Over the past year, we have pointed to the benefits of our commercial head start and the foundation we built with ZYNTEGLO for beta-thalassemia and SKYSONA for cerebral adrenoleukodystrophy. Today, we are seeing not only that momentum build for our first two launches, but also these synergies are coming to fruition for LYFGENIA. As a reminder, our launches are focused on three core elements for success. First, establishing a robust network of qualified treatment centers or QTCs. These are transplant centers with significant experience in cell and gene therapy. Today, we have 64 activated QTCs, unparalleled compared to others in the field. Second, ensuring the value of our therapies is recognized and that patients have timely, equitable access. And lastly, optimizing the patient and provider experience. Gene therapy requires a high-touch approach and transparency, collaboration, and understanding the needs of patients, families, and providers is paramount. Our deep understanding of the gene therapy process, our dedicated focus, and our experience over the last 18 months allow us to be a strong partner to our activated QTCs as we help them bring life-changing therapies to their patients. We are extremely encouraged by the excitement in the sickle cell community and the number of patients preparing for treatment. As we sit here today, more than half of our LYFGENIA QTCs have communicated to us that they are actively evaluating patients for gene therapy initiation, and one quarter of our centers are in the prior authorization process for one or more patients. As a reminder, the journey to a patient start or cell collection takes time. While every patient is different, the typical path for a patient to first initiate a consultation with a transplanter at a QTC, then to enroll in myBluebird support, and then often concurrently, the QTC will initiate reimbursement negotiations with the payer, while also taking steps to ensure clinical readiness, which includes a two-month washout period for hydroxyurea. These steps must be coordinated, and the patient's health must be considered. With that in mind, we continue to anticipate starts for LYFGENIA to grow quarter over quarter, with the majority occurring in the second half of the year as momentum builds. Factoring in the time for drug product manufacturing once the patient cells are collected, we anticipate that first revenues for LYFGENIA will be recorded, reported in Q3. Moving to ZYNTEGLO, we continue to see strong linear growth with 11 patient starts since the beginning of 2024. As we previously shared in mid-2023, we initiated steps to increase drug product manufacturing capacity for ZYNTEGLO and SKYSONA, commensurate with demand. Through our experience in the market, we've also determined the need to increase adherent vector supply, and we've initiated steps to bolster supply to meet the launch trajectory. Additionally, we have completed 3 patient starts for SKYSONA since the beginning of 2024. As a reminder, patient starts, which is when cell collection occurs, remain the key commercial metric to watch in the early stages of our launch, as this is the value-creating moment for the company, with revenue being recognized when the patient is infused. For modeling purposes, you can continue to expect that patients are infused 1-2 quarters following cell collection. In our experience, once a patient goes through cell collection, they continue on the treatment journey and are dedicated. To date, every patient who has started the process has completed or remained in the process, and we continue to expect between 85 and 105 patient starts across our commercial portfolio in 2024. Now moving to access and reimbursement. Our validated strategy from our ZYNTEGLO and SKYSONA experience is driving favorable coverage for LYFGENIA. We are very pleased to have our cost effectiveness model for LYFGENIA peer-reviewed and published in the Journal of PharmacoEconomics in April. The publication offers significant insight into the potential lifetime impact that reducing or eliminating VOE-associated pain crises may have on patients' health and well-being, healthcare utilization, future earnings, and life opportunities. The publication found that LYFGENIA is cost-effective up to a price of $3.9 million. The value is being recognized, and we are making great progress securing access for patients with sickle cell disease. Just five months post-launch, both commercial and Medicaid-insured patients have successfully obtained prior authorization for LYFGENIA. Additionally, we have multiple outcomes-based agreements signed for LYFGENIA with national commercial payer organizations and have published coverage policies in place for more than 200 million U.S. lives. Our goal has always been timely, equitable access to LYFGENIA, irrespective of a patient's insurance type. Discussions are ongoing with Medicaid agencies, representing 80% of Medicaid-insured individuals with sickle cell disease in the U.S. Additionally, timely access to ZYNTEGLO and SKYSONA has continued, with zero ultimate denials for either therapy across both Medicaid and commercial payers. Moving to our QTC network, we have established a substantial QTC footprint very quickly following LYFGENIA's approval. Today, Bluebird has activated 64 QTCs for LYFGENIA and ZYNTEGLO. We were able to quickly transition these centers for LYFGENIA due to both the learned experience of setting up these centers for ZYNTEGLO and the strong relationships we've built with these centers during 2023. Eleven QTCs have started their first patient for one of our products, and the majority of sites who have started their first patient have started their second or third. While we anticipate we may bring on a handful of additional centers throughout 2024, our focus has shifted to patient pull-through at our 64 centers and deepening their experience with bluebird's gene therapy portfolio. Additionally, six centers in our network are also activated to administer SKYSONA for patients with CALD. To recap, ZYNTEGLO and SKYSONA launches continue to progress as planned, and we anticipate strong linear growth for ZYNTEGLO in 2024, along with 5-10 patient starts for SKYSONA. We are extremely excited about the early progress in our LYFGENIA launch, building on our validated commercial platform, and most importantly, gene therapy is becoming a reality for patients in the United States. We look forward to updating you as our momentum builds in our launches and patient starts to grow over the next few quarters. Now I'd like to turn the call over to Chris. Thanks, Tom, and good morning, everyone. In the first quarter, we reported $18.6 million in total revenue, driven by revenue from ZYNTEGLO. As a reminder, we recognize revenue upon infusion of the drug product. As previously guided, in 2024, we anticipate gross-to-net discounts in the range of 20%-25%, with fluctuations based on product mix, payer mix, as well as utilization of our outcomes-based agreements. As of March 31, 2024, we had $264 million in cash on hand, inclusive of $52 million in restricted cash. Based on our current business plans, including our ability to achieve certain commercial revenue milestones, we have a cash runway through Q1 of 2026. This runway includes our current cash equivalents and also assumes that we receive the two remaining tranches, totaling $50 million from our term loan facility. Additionally, we continue to work expeditiously to complete our restatement and file our 2023 10-K and Q1 2024 10-Q. I want to personally thank and recognize the tremendous work and unwavering dedication of the bluebird finance and accounting teams that are finalizing the restatement. Importantly, and as a reminder, the restatement is not expected to impact our cash position or our revenue. With that, I'll turn it back over to Andrew. Thanks, Chris. As we highlighted today, it's an exciting time for Bluebird. We stand in the midst of a monumental year with the potential for growth on the near-term horizon. With that, I'd like to open it up for questions. Operator? Thank you. As mentioned, the floor is now open for questions. To ask a question, please press star one on your telephone keypad to raise your hand and join the queue. In the interest of time and to ensure we cover as many participants as possible, we do request a limit of one question and one follow-up per person. Your first question comes from the line of Jason Gerberry from Bank of America. Your line is open. Morning. Thanks for taking my question, guys. I guess I'll ask a question about just the update on the Medicaid reimbursement work that's ongoing. I'm curious, I think we asked this question last quarter, but just wanted to make sure that it's not a rate-limiting factor at all to new patient starts for LYFGENIA. ... as you build up the Medicaid reimbursement coverage. And just trying to put that in context relative to your competitors, update on their new starts. I guess the question is one of, are they launching faster in the U.S. or is their new patient start number more just a function of a broader geographical reach? Yeah. Morning, Jason. I'll hand that over to Tom. Why don't we take those in order, kind of, reverse order? Why don't we talk about the patient starts first, and then talk about the Medicaid as well? Go ahead. Yeah. Good morning, Jason. We're extremely pleased with what we're seeing in terms of starts. Obviously, we have a tremendous head start with ZYNTEGLO and a lot of progress there. But when you look at LYFGENIA, we have multiple patients in multiple QTCs going through the initiation process for gene therapy, and very excited about the first collection. We're really not gonna comment about, you know, what the competitor is doing, but, you know, more so I think we feel really good about what we're seeing with LYFGENIA. As far as Medicaid, it's not a rate limiting factor. Obviously, we've been working with Medicaid for many, many years now on making sure they understand and recognize the value of a one-time potentially curative therapy. Also, offering our outcomes-based agreement and tying that to the value of our therapy. In the meantime, until they have coverage policies or, you know, sign up for an outcomes-based agreement, they can always have access. Basically, patients can have access through the medical exceptions process, so it's really not a barrier for patients. Got it. Thank you. Your next question comes from the line of Jack Allen from Baird. Please go ahead. All right, thank you so much for taking my question. I guess, to start, I wanted to ask about your your thoughts as it relates to studying LYFGENIA in younger patients. Do you have a study ongoing, and how are things progressing as it relates to expanding access, as it relates to age for LYFGENIA? Yeah, thanks for the question, Jack. So, we have a trial on HGB-210 ongoing in pediatric patients. That enrollment's ongoing. We anticipate to complete that by Q4, 2024. And that's gonna evaluate patients 2 to 12, for LYFGENIA. As a reminder, for ZYNTEGLO, we have an indication down to the age of, of 2. Got it. Great. And then I'll give it a shot here. I'm not sure what kind of reception I'll get on this, but how many patients are in the bluebird support system as it relates to LYFGENIA? Do you have any idea about the pull-through of those patients based on your ZYNTEGLO experience? Is there anything to read through there as it relates to people starting that aspect of the process? Yeah, good question, Jack. You know, we've learned a lot through our ZYNTEGLO experience. We're not gonna comment individually on the pull-through because I think, you know, when you look at beta-thalassemia, it's a lot different than patients who have sickle cell disease. And it's kinda early in the process right now with obviously a small N for LYFGENIA, so we really don't wanna comment on that. But I will tell you that there are a lot of patients who are very excited for gene therapy. As I said, there are multiple patients across multiple QTCs initiating the process, and usually once they start the process, they're committed and continue through the process. We're just excited with the momentum we're seeing, and we look forward to, you know, getting more data as the year progresses. Great. Thanks so much for taking the questions. Your next question comes from the line of Daniel Brill from Raymond James. Please go ahead. Morning, this is Daniel Ong for Daniel. We have a question on the QTC is qualified for both Casgevy and LYFGENIA. Any particular reason for the patient chose to pursue the treatment with LYFGENIA? And have you seen any, like, push and pull from payers deciding either therapy? Thank you. Yeah, thanks for the question. Tom, go ahead. Yeah, good morning, Daniel. As far as offering both therapies, we have a head start, obviously, with 64 Qualified Treatment Centers. However, it is our expectation that most Qualified Treatment Centers will offer both therapies, LYFGENIA and the other gene therapy. We haven't seen many centers saying they're gonna offer one or the other or exclude one or the other. I think most centers believe that choices for patients are good, and if they have both onboarded already, which is only a small handful, then they present both to the patient, and it becomes a patient choice. Thank you. Your next question comes from the line of Gena Wang from Barclays. Please go ahead. Thank you for taking my questions. Maybe also asking about the 64 QTC. Out of these, how many of these actually were active for LYFGENIA? And also for the first patient, I don't know if you can share that detail, how many cycles of the cells collection in order to start the manufacturing? Yeah. Morning, Gena, and actually, I think I'll take the second part of that first and hand it over to Tom for the first part. That was the first collection for the patient. We reported their first collection, so we haven't reported whether they how many collections we'll need, but manufacturing is ongoing right now, so we can't comment on that. Tom, go ahead. Yeah. Hi, Gena. The 64 centers, we're really excited about that. Centers really do not wanna go through the process of getting onboarded if they don't have patients to treat. So we believe that the 64 centers that we have, you know, will evaluate patients for LYFGENIA and/or ZYNTEGLO as we go forward. And if you look at the dynamics of how we build out the Qualified Treatment Center, almost half of the Qualified Treatment Centers have come on board just since approval in December. So the momentum continues to build, and as you look at the patients going through the centers, we're really excited that 64 centers are now on board, and many of them are already starting the process of evaluating patients. ... I'm sorry, if I can make it clear, I think last quarter was 49 out of 62 received referral for LYFGENIA. So just wanted to know now, 64 out of 64, how many of these received referral for LYFGENIA? Yeah, it's about 50 of the 64, and we anticipate that almost all of them will be on board very soon. It's just a matter of completing some final steps. And in some cases, you know, you won't see all of them on our website, because some of them have chose not to be listed on our website. Okay, great. Thank you. Your next question comes from the line of Eric Joseph from JP Morgan. Please go ahead. Thanks, good morning. The added color that you're providing here on sort of product collections within the QTC network is pretty interesting. Can you talk a little bit more about sort of what separates the initial 11 QTCs that have treated patients so far versus the balance? And I wonder whether it's perhaps bed capacity or patient demand or something in the pre-authorization process that has the first group kind of moving a little more swiftly. And then, as a follow-up to this Times article featuring your first patient collection with LYFGENIA. The article notes that Children's National has a capacity of about 10 to do about 10 gene therapy treatments a year. Can you talk about how representative that capacity is across your QTC network? Do you anticipate any efforts to kind of expand that in a way that would support uptake? Thank you. Go ahead, Tom. Yeah, good morning, Eric. Two great questions. The first question, probably I can answer that two different ways. I think if you consider the dynamics of when we brought on the QTCs, you know, we really focused on the first 10-15 for the most, you know, the first half of last year, and then we rapidly accelerated into the end of last year and the start of this year. So if you look at, you know, those initial 10-15, now you're seeing roughly 11 are treating at least one patient or going on to treat their second and third patient. The second component is just time. If you look at the sickle cell, the patient journey for sickle cell disease, it just takes time from, you know, the initial consultation, going back and discussing it with their family, going through the process of getting the prior authorization, and then being medically ready. You know, making sure they're stopping their hydroxyurea for two months and going through transfusions. I would say the early adopters kind of saw this in advance and were thinking about patients, and patients were already calling in prior to approval, and that's why you're seeing some QTCs get off to a faster start. And the second part of your question around the New York Times article, very inspiring to see, you know, that patient go through the start of the process. I would say if you've seen one Qualified Treatment Center, you've seen one Qualified Treatment Center. So I wouldn't say that that's necessarily representative, but I would say that most of the large QTCs are pulling out, you know, large capacities for cell and gene therapy. Okay, thanks. Maybe just a quick follow-up to that. Did Children's National or has Children's National treated any patients with ZYNTEGLO? We are not going to comment individually by QTC by QTC on that information, but, but, I think they're obviously one of the 11 that's treated patients, so and many of those QTCs have now gone on to their second and third, so I'll just say it that way. Okay. Thank you very much. Your next question comes from the line of Eric Schmidt from Cantor Fitzgerald. Please go ahead. Thanks for taking my questions. In terms of the new start guidance for 2024, just wanna maybe put a finer point on how you're quantifying a new start or qualifying a new start. Is it from the time of the patient's first collection has been completed, does that make them a new start? Go ahead, Tom. Yeah, good morning, Eric. So we've been pretty consistent in how we've defined new patient starts, you know, since the beginning. We really believe that the metric to watch is the first unique cell collection. When the cell collection is complete, we believe that once a patient goes through that process, that it's highly likely that they'll go through the entire journey and ultimately get treated and infused at the end of the day. So, we define it by that unique first cell collection being completed. Thanks for that information. Maybe, maybe for Chris, you know, we're kind of flying into uncharted waters with regard to the restatement. Is there anything you can say on how COGS are trending, OpEx, cash burn in Q1? I guess I'm struggling with being able to understand your financial state right now. Go ahead, Chris. First, I'll say that the restatement will not have an impact on cash, nor it will have an impact on the key metrics for the company, such as patient starts, et cetera. That's why we continue to make that message. I'm not gonna comment on the other elements of the lines of the P&L associated with where we're trending, just because we're in the process of the restatement, and it's not fair or appropriate to comment on that. Do you have a better sense on when we'll see those numbers? We have the skills that we're working expeditiously to complete the restatement. We're not providing a timeline associated with when that will occur. Thank you. Your next question comes from the line of Mani Foroohar from Leerink Partners. Please go ahead. Hey, guys, this is Ryan on for Mani. Thanks for taking our question. Kind of a two-parter one. We were just wondering, you know, how should we think about the pace of cell collections for LYFGENIA as we go through the year? Are we gonna start to reach a, you know, a steady state at some point? Do we see this accelerating into the end of the year? And then, you know, kind of dovetail off of that, can you guys talk about, you know, what specific actions your field team is taking to kind of help accelerate the LYFGENIA launch, whether it's through QTC support, you know, engaging with physicians, et cetera? Thanks. ... Yep, go ahead, Tom. Yeah. Good morning, Ryan. We really have—we've said all along, we expect the trajectory of our starts to really accelerate and ramp into the end of the year in Q3 and Q4. And the field team has shifted their focus completely from bringing new qualified treatment centers on board, to now focusing on supporting qualified treatment centers in pulling through patients and making sure that they're ready to pull through patients. So we're completely shifting gears. Our field team is, you know, has built great relationships with the qualified treatment centers over the last year and a half. You know, a lot of credibility and rapport, but now completely focused on supporting patient pull-through. Awesome. Thank you. Your next question comes from the line of Yannan Xu from Wells Fargo. Please go ahead. Hi, thanks for taking our question. This is Kuan Ang for Yanna. So first, a quick question on with Jenny. So any colors on how many patients have started their hydroxyurea washout? I think the question was, how many patients have started the hydroxyurea washout, Tom? Yeah, so it, it varies. We weren't going to really give guidance on that. It varies by not only patient, but by Qualified Treatment Center. Some patients are on Hydroxyurea, some patients are not. The Qualified Treatment Centers have different SOPs in terms of when they start that washout period. Obviously, you know, they wanna start some of the medical readiness concurrently with some of the prior authorization work that they need to do. And that's pretty consistent, but we aren't giving guidance on kinda some of the precursors before the cell collection. Got it. Thank you. One quick question on manufacturing. What's your current capacity at CDMO for LYFGENIA? And is there any additional investment needed if you want to increase the capacity in the future? Thank you. Yep. So I think the question about capacity for LYFGENIA, and I think it was probably talking about drug product capacity. So we do have a separate supply chain for LYFGENIA, that is ZYNTEGLO and SKYSONA, that we do have more robust supply on the LYFGENIA side in anticipation of a higher volume. We also have the ability to increase that supply as we see demand come in. So we're gonna match how we bring on new capacity with how we see the demand come in. So there is, as close to lockstep as possible. Got it. Thank you so much. Your next question comes from the line of Luca Issi from RBC. Please go ahead. Oh, great! Thanks for taking our question. This is Lisa on for Luca. Just wondering if you can talk about whether you are reconsidering launching in ex-US geographies. For instance, your competitor seems really excited about the Middle Eastern market, stating there are potentially 23,000 eligible patients in that region. So wondering if you are reconsidering an ex-US launch, either solo, with a partner, or utilizing a distributor. Any thoughts there are much appreciated and congrats on all the progress. Thanks. Yeah, thanks very much. So right now we are entirely focused on the U.S., and we will remain focused on the U.S. for the very, at least near and probably midterm, future. We are watching the ex-U.S. geographies. If we go there, we'd most likely go with a partner and not do it alone. Thanks. Got it. Your next question comes from the line of Sami Corwin from William Blair. Please go ahead. Hi, this is Brooke Schuster on for Sami. Thank you for taking our question. So with a total of 64 QTC centers activated, I guess, what would you say is the biggest bottleneck to the initiation of patient starts? And we are also wondering if some of the ZYNTEGLO patients that had cells collected in Q4 have, or if all of them been treated already. Thank you. Go, Tom. Yeah. Hi, good morning. So it's, I wouldn't call it a bottleneck. I would just characterize it as time. It just takes time for patients to go through the process. You know, if you look at sickle cell disease, one big difference from sickle cell to beta-thalassemia. With beta-thalassemia, patients are already basically medically ready. They're going through regular red blood cell transfusions and, you know, are identifiable and mostly ready to go for treatment. When it comes to sickle cell disease, many of these patients are not already in a QTC. Many of them are not doing transfusions currently, and some of them are on hydroxyurea. So from a medical readiness perspective, it just takes a little longer for a patient to be ready to go through the treatment process for LYFGENIA. The insurance process obviously takes a little bit of time as well, and, you know, that starts with the first patient, and the first patient usually takes a little bit longer until the QTCs get into a cadence and the payers start to understand more about the cadence of the QTCs. So it's really not a bottleneck per se, it just takes time. Your next question comes from the line of Salveen Richter from Goldman Sachs. Please go ahead. Hi, this is Lydia on for Salveen. Thanks so much for taking our question. Could you just discuss your confidence in achieving the patient start guidance this year? And do you still intend for roughly half of these patients to come from LYFGENIA? Thanks so much. Go ahead, Tom. Yeah, we're pleased with how the launches are going. Obviously, a lot of momentum building with ZYNTEGLO. We feel that there is consistency with what we're seeing with SKYSONA, and then with 64 qualified treatment centers, we're hearing a lot of positive experience in terms of patients excited about gene therapy and patients ready for gene therapy. So based on not only what we did kinda pre-launch and the numbers that we're running, but also based on what we're hearing in the field today, we remain confident in the 85-105 starts this year, and obviously, that will ramp in the second half of the year as the treatment centers get through the process. This does conclude our Q&A session for today. I would like to hand the call back over to Andrew for closing remarks. Thank you. Thanks, everyone, for joining our call this morning and for your questions. Our management team is available for follow-up calls today, and please reach out to Courtney if you would like to connect. Thank you. This concludes today's conference call. Enjoy the rest of your day. You may now disconnect.