Editas Medicine, Inc.

Good morning and welcome to Editas Medicine's fourth quarter and full year 2023 conference call. All participants are now in a listen-only mode. There will be a question and answer session at the end of this call. Please be advised that this call is being recorded at the company's request. I would now like to turn the call over to Christy Barnett, Corporate Communications and Investor Relations at Editas Medicine.

Thank you, Maria. Good morning, everyone, and welcome to our fourth quarter and full year 2023 conference call. Earlier this morning, we issued a press release providing our financial results and recent corporate updates. A replay of today's call will be available in the investors section of our website approximately two hours after its completion. After our prepared remarks, we will open the call for Q&A. As a reminder, various remarks that we make during this call about the company's future expectations, plans, and prospects constitute forward-looking statements for the purposes of the safe harbor provisions under the Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors including those discussed in the risk factors section of our most recent annual report on Form 10-K, which is on file with the SEC as updated by our subsequent filing. In addition, any forward-looking statements represent our views only as of today and should not be relied upon as representing our views as of any subsequent date, except as required by law, we specifically disclaim any obligation to update or revise any forward-looking statements even if our views change. Now, I will turn the call over to our CEO, Gilmore O'Neill.

Thanks, Christy, and good morning, everyone. Thank you for joining us today on Editas' fourth quarter and full year 2023 earnings call. I am joined today by four other members of the Editas executive team, our Chief Medical Officer, Baesong Mai, our Chief Financial Officer, Eric Lucera, our Chief Scientific Officer, Linda Barkley, and our Chief Commercial and Strategy Officer, Karen Dierdorf. We are pleased with Editas' momentum and progress in the fourth quarter and all of 2023. In early 2023, we shared our vision and the three pillars of our strategy to position Editas as a leader in in vivo, programmable gene editing, and hemoglobinopathies. The first of these pillars to drive Renicel, formerly known as Edit 301, toward BLA and commercialization. The second, to strengthen, reorganize, and focus our discovery organization to build an in vivo editing pipeline. And the third, to increase business development activities with a particular focus on monetizing our very strong IP. So how did we do last year? Well, we achieved a lot. First, we accelerated the clinical development of Renicel, exceeding our enrollment goal of 20 patients and sharing clinical updates from our Ruby and Edital studies in June and in December of 2023. Those accumulating data have strengthened our belief that Renicel is a competitive potential medicine with a differentiated profile characterized by correction of anemia and the normal physiologic ranges of hemoglobin. Second, we strengthened our in vivo discovery capabilities and organization and hired a new chief scientific officer, Linda Berkley, who brings three decades of experience in successfully inventing, developing, and moving new human medicine forward. And third, We increased our business development activities and monetized our IP, leveraging our robust IP portfolio. A critical example was our granting Vertex a non-exclusive license for our CAS 9 IP in a focused way to enable the XSL launch. Finally, we strengthened our senior leadership team with people who have a proven track record in bringing new medicines through development to approval and commercialization. So how have we executed against these strategies? and these objectives. Well, let's start with RaniCell. First, on enrollment, we have now enrolled 40 clinical cells and nine beta thalassemia patients in our Ruby and Edithal studies, respectively, and enrollment continues at a good pace. Second, on dosing, we have dosed 18 Ruby patients and seven Edithal patients, and we have multiple patients scheduled for dosing in the coming months. Patient screening and demand in both studies continue to remain robust. Third, on clinical data, We remain on track to present a substantive clinical data set of sickle cell patients with considerable clinical follow-up in the RUBI study in the middle of 2024, with a further update by year-end 2024. On the regulatory front, we have engaged with the FDA regarding the RUBI sickle cell trial. The FDA agrees that RUBI is a single phase 1, 2, 3 study and has aligned with us on the study design. Our discussions with the FDA will continue as RUBI and EDIthel progress. and will be enhanced by our RMAF designation for severe sickle cell disease. Besson will share further details regarding the development of Renicel in his remarks, as well as recap the Ruby and Edifal takeaways and clinical data that we provided in December, and share more information on the adolescent cohort. Now, let's turn to in vivo and our pipeline development, where we strengthened our in vivo discovery capabilities in 2023, and began lead discovery work on in vivo therapeutic targets in hematopoietic stem cells and other tissues. As we announced earlier this year, we aim to establish in vivo preclinical proof of concept for an undisclosed indication this year. Linda and her team are leveraging key capabilities that we have in-house and should forward to sharing more at a future date. Regarding our hemoglobinopathy focus, After a thorough evaluation of the development landscape, we have decided not to pursue internal development of a milder conditioning regime. We believe standalone milder conditioning regimens would be widely available once FDA approved, and therefore, we have determined that research, clinical development, and regulatory investment in hemoglobinopolis can be better deployed for our continued development of in vivo HSC methods. Turning to business development, in the fourth quarter, we announced a license agreement with Vertex Pharmaceuticals. Editas provided Vertex a non-exclusive license for Editas Medicine's Cas9 gene-editing technology for ex vivo gene-editing medicines targeting the BC11A gene in the field of sickle cell disease and beta-palatemia, including Vertex's CasGV. And the upfront and contingent payments pursuant to this agreement extended our cash runway into 2026. This and other agreements The strength of our patents and the number of companies developing CRISPR-Cas9 medicine reaffirm our confidence that our IP portfolio of foundational U.S. and international patents covering Cas9 use in human medicine are a source of meaningful value. So what are our objectives for 2024? For Renicel, we will provide a clinical update from the Ruby trial for severe sickle cell disease and the Edifal trial for transfusion-dependent beta-thalassemia in mid-2024 and by year-end 2024. We will complete adult cohort enrollment and initiate the adolescent cohort in Ruby, which we've already initiated and continue enrollment in ASL. For our in vivo pipeline, we will establish in vivo preclinical proof of concept for an undisclosed indication. And for BD, we will leverage our robust IP portfolio and business development to drive value and compliment core gene editing technology capabilities. We are energized by our progress and execution in 2023. With our sharp and strategic focus, our world-class scientists and employees, and our keen drive and execution, we continue to build momentum to progress our strategy to deliver differentiated editing medicines to patients with serious genetic diseases. Now, I will turn the call over to Beisong, our Chief Medical Officer.