Editas Medicine, Inc.

Good morning and welcome to the Editas Medicine first quarter 2024 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. Good morning, everyone, and welcome to our first quarter 2024 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 filings. 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' first quarter 2024 earnings call. With me today are four members of the EDItas executive team, our chief medical officer, Baesong Mai, our chief scientific officer, Linda Berkley, our chief financial officer, Eric Gucera, and our chief commercial and strategy officer, Karen Dierdorf. We are pleased with EDItas' momentum and progress in the first quarter of 2024. EDITAS's goal is to deliver life-changing medicines to patients with previously untreatable or undertreated genetically determined diseases. And our vision and focus strategy is to position EDITAS as a leader in in vivo programmable gene editing. Three pillars underpin our strategy. The first of those pillars is to drive RENI cells and edited cell therapy for hemoglobinopathies and formerly known as EDIT301 toward BLA and commercialization. The second is to build an in vivo editing pipeline. And the third is to increase business development activities with a particular focus on monetizing our very strong intellectual property. At the start of 2024, we announced the following 2024 objectives. For ReniCell, we will provide a clinical update from the Ruby trial for severe sickle cell disease and the Edithal 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 and continue enrollment in EDITAL. 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 complement core gene editing technology capabilities. So, how we executed against this strategy and these objectives in the first quarter. Let us start with Renicel. First, on enrollment. We've been very pleased with the growing patient and healthcare provider interest in Renicel. Indeed, we are delighted to share that we have completed enrollment in the adult cohort of the Ruby clinical trial. Additionally, we have enrolled multiple patients and have more in screening in the adolescent cohort of our Ruby study, which was launched at the beginning of this year. and we continue to enroll beta thalassemia patients in our Edithal study. Dosing continues in both the Ruby and Edithal studies. Second, on clinical data, we remain on track to present a substantive clinical data set of at least 18 sickle cell patients with two to 21 months of clinical follow-up in the Ruby study in the middle of 2024, and we will share a further update by year end. We are also on track to present clinical data from the Edithal study of Renicel in transfusion-dependent beta thalassemia in the middle of 2024 and again by year end. Bae-Song Mai will share more Renicel data later on in this call. On the manufacturing front, I am pleased to share that we have promoted Greg Whitehead to the role of Chief Technology and Quality Officer, leading our technical development, technical operations, and quality departments. Greg has more than 25 years of experience in the biotech industry and extensive cell and gene therapy clinical and commercial development expertise. Now, let's turn to in vivo and our pipeline development, where we continue to strengthen our in vivo discovery capabilities and continue lead discovery work on in vivo therapeutic targets in hematopoietic stem cells and other tissues. Importantly, we remain on track to establish in vivo preclinical proof of concept than undisclosed indication by the end of the year. Our internal development efforts are differentiated by leveraging the INDEL CRISPR technology we already use to upregulate gamma-globin expression through direct editing of the HBG1-2 promoter site in our ex vivo ready cell program. Our in vivo approach is aimed at functional upregulation of gene expression in genetically defined diseases with a preliminary focus on rare and orphan patient populations. In the medium to long term, we intend to expand to more common genetically determined diseases. Linda Berkley, our CSO, will share more details on our in vivo strategy and progress towards building an in vivo pipeline later on in the call. Finally, what is happening in business development? In March, we signed a two-year extension to the collaboration with Bristol-Myers Squibb to research, develop, and commercialize autologous and allogeneic alpha-beta T-cell medicines for the treatment of cancer and autoimmune diseases. We also have options to extend that collaboration for an additional two years. To date, Bristol-Myers Squibb has opted into 13 different programs across 11 gene targets to date. Two programs are currently in IND-enabling studies, and four programs are in late-stage discovery. And in intellectual property, yesterday, oral arguments were held before the U.S. Court of Appeals for the Federal Circus regarding an appeal of the Patent, Trial, and Appeal Boards, or PTABs, previous decision favoring Broad Institute in the U.S. patent interference involving specific patents for CRISPR-Cas9 editing in human cells between the University of California, University of Vienna, and Emmanuel Charpentier, or CDC, and the Broad. We expect a decision on the case in the second half of 2024. Eric will share more BD and IP details later on in the call. We are energized by our progress in execution this quarter, with our sharpened strategic focus our world-class scientists and employees, our keen drive and execution, and strong balance sheets, we continue to build momentum to progress our strategy to deliver differentiated editing medicines to patients with serious genetic diseases. Now, I would turn the call over to Bei-Tang, our Chief Medical Officer.