bluebird bio, Inc.

Our CEO is going to provide some brief opening remarks and discuss our Lobosol BLA submission. Then Tom Klimak, Chief Commercial and Operating Officer, will highlight positive momentum from our Zanteglo and SkySona commercial launches. And finally, Chris Krawchuk, our Chief Financial Officer, will provide some color on our finances before opening the call-up for Q&A, where the team will be joined by Rich Colvin, Chief Medical Officer. With that, I will turn the call over to Andrew.

Thanks, Courtney. And thank you everyone on the line for joining the call this morning. Bluebird's vision and mission are stronger than ever. Built on a strong foundation of research and clinical development, today Bluebird is making gene therapy a reality for patients and families in the real world. And I'm immensely proud of the efforts of our entire company as we continue to lead the way for the gene therapy field. Proving the commercial model for ex vivo gene therapy with Zyntegra and Spisona and continuing to make meaningful progress towards bringing gene therapies to patients and their families with sickle cell disease in the U.S. As Courtney mentioned, Tom and Chris will share updates on our commercial progress and our financial position. But to start, I will focus my comments on the Lovis L regulatory path. As noted in our press release this morning, we continue to progress our Lovis L BLA, but speaking plainly, we will likely miss the Q1 2023 submission goal. Our file is completely written and ready for submission, but we are awaiting feedback from the FDA on CMC. To provide context, timeline of our recent interactions with the FDA. In December, we completed drug products comparability analyses and provided the FDA with a snapshot of comparability data. In February, we received feedback and questions from the FDA and a request that these responses be provided prior to the submission of the BLA. Our team did incredible work and pulled forward the full CMC module along with additional information and provided them to the agency in the first week of March. We believe that this complete data package supports comparability and addresses the questions asked. Now, while there's no set timeline for the FDA to respond, the agency has conveyed its commitment to a timely response, and we believe that this could ultimately set the stage for a smooth review. We anticipate feedback from the FDA within a matter of weeks, and we'll move quickly to expedite our BLA pending the resolution of the comparability questions. And I will reiterate, the file is otherwise complete and ready for submission. We are grateful to the FDA for the ongoing open dialogue and appreciate the importance of ensuring the agency understands the full contents of our analyses. To take a step back, I'd like to remind everyone of the depth of preparation that has gone into both the manufacturing and the clinical development of LovaCell and the work that will be reflected in the BLA. First, manufacturing. CMC modules are the largest sections in any gene therapy BLA. With that in mind, Bluebird has had an active and collaborative dialogue with the FDA on comparability over the past several years. We've also incorporated learnings from Skysonins and Tegla into our BLA for LoboCell. Now, as a reminder for those newer to the Bluebird story, through the course of clinical development for LoboCell, we made improvements in the manufacturing of our lentiviral vector and of our LoboCell drug product, including changing manufacturing processes, manufacturing facilities, and testing sites. This included changing from an adherence to a suspension process to ensure that we would meet the patient demand at launch with a robust, scalable, and commercially compliant process for the 20,000 patients we believe may be eligible for gene therapy. Now, some of these changes occurred following the completion of our HDB206 study, which forms the primary evidence base for safety and efficacy to support the BLA. Therefore, the industry required that we demonstrate vector and drug product comparability, where we collect and analyze all the manufacturing data generated from our commercial processes in our commercial facilities and compare them to the manufacturing data generated from our clinical trials in our clinical facilities. These analyses were completed at the end of last year. We remain extremely confident in the quality of our overall BLA submission package. And as part of that, let me move to our impressive clinical data. Our clinical data reflects the most robust data available with the longest follow-up across any gene therapy program for sickle cell disease. And it includes more than 50 patients treated and multiple patients followed for more than six years. The strength of this package has been reinforced by positive interactions with the FDA. The BLA submission will be based on efficacy results from 36 patients in the HGB 206 Group C cohort. It will include a median of 32 months of follow-up and more than four and a half years of follow-up for some patients. We anticipate that the BLA submission will also include efficacy results from two patients with 18 months of follow-up in the HGB210 study. We plan to request priority review for patients 12 and older with a history of vaso-occlusive events. Importantly, at this time, we are not forecasting any change to our commercial plans for Lopacil and are continuing to anticipate an early 2024 launch. And we are really looking forward to delivering on the promise of gene therapy for sickle cell patients who have been waiting for therapies like this. As Tom will discuss, the foundation that we are building today to bring Zantaglo to patients will directly translate into our ability to bring LogoCell to patients. We will have the same treating physicians, the same QTC network, and the same pay relationships. Bluebird has over a year ahead start launching a gene therapy in inherited hemoglobin disorders versus any other gene therapy program. And with that, I will turn it over to Tom to highlight how our launches are progressing.

Thanks, Andrew, and good morning, everyone. It's exciting to be here this morning to discuss the strong momentum we've built for our two approved gene therapies, Zynteglo and SkySun. There's a lot of attention and noise in gene therapy these days, But the reality is that at Bluebird, we are living it every day and bringing these important one-time treatments to patients. During a recent field visit, I received feedback from healthcare professionals in our QTCs. They praised Bluebird for our continued partnership and for leading the way with our deep expertise and through transparency. They reinforced that our trusted partnership over the years will be an important differentiator. As a reminder, our commercial strategy is built on three key pillars which are fundamental to success in gene therapy launches. First, patients and their families are at the center of everything we do, and they are the ultimate decision maker. Our continued engagement over the past decade and our ongoing education about gene therapy are critical for adoption. Second, and unique to Ex vivo gene therapies, we're building a network of qualified treatment centers, or QTCs, where the cell collection and infusion of our therapies takes place. These centers of excellence are both part of our supply chain, but they're also ambassadors for patient care and ultimately for our therapies. And finally, access and reimbursement, an area where we have innovated as pricing and securing coverage for a one-time therapy is certainly not the same as pricing a chronic therapy. Importantly, we are seeing success in all three areas since launch, with momentum continuing to build. For Zynteglo, we are seeing significant patient demand and uptake, highlighting the tremendous unmet need and interest in gene therapy from patients with beta thalassemia. To date, there have been five patient starts or cell collections for patients with beta thalassemia coming from our early wave one QTCs. This momentum will continue to build as we grow our QTC network. And notably, as launch progresses, we are already advancing our plans to expand our manufacturing capacity to meet growing projected demand. On the reimbursement front, we're pleased to report that things continue to go very well. On average, prior authorization is taking only two weeks, a strong indicator that payers recognize the value of Zynteglo in a rare disease with significant unmet medical need. Most important, we continue to see zero ultimate denials. Switching to QTCs, we've expanded our QTC network as planned with 12 centers activated to date, representing a mix of both pediatric and adult centers. Approximately 30 additional QTCs are in the onboarding stage, or MSA negotiation phase, and the company remains on track to scale to between 40 and 50 QTCs by the end of 2023. This progress is because of our preparation and is also a signal of patient interest and enthusiasm among physicians for ex vivo LDV gene therapy. Importantly, as Andrew mentioned, the efforts we're making with Zenteglo will directly translate to the potential launch of low cell for sickle cell disease in three key ways. First, and very simply, the treating physicians are the same. Second, the Bluebird QTC network will be the same. This means that from an operational standpoint, our expectation is that we will reduce the lead time for QTC activation from months to weeks because of the synergies. We're really doing the groundwork now so that we will be ready to begin treating patients shortly after approval. And lastly, with payers, we're already being recognized as leading the way with our approach to value demonstration and innovation with our outcomes-based agreements, and this established credibility will carry forward. We will continue to provide updates on key metrics from the Zyntegra launch, including number of patient starts as launch progresses. We believe the most important metric for you to keep an eye on is the number of patient starts. For SkySona, cell collection has been completed for two patients. And on March 16th, the first commercial infusion was completed at Boston Children's Hospital. I cannot overstate what an incredible moment this was for this patient and his family, for the clinicians and researchers, and for the entire ALD community. CALD is a devastating disease, and this was a milestone celebrated by all those who have advocated, invested, and fought for treatment options for many, many years. It is difficult to summarize the passion and the pride we all have in being able to deliver SkySona to that young boy and his family. In conclusion, I'm extremely proud of the progress and dedication of our team and of how we're leading the way as a commercial gene therapy company. With that, I'll turn it over to Chris to talk through the financials.