Editas Medicine, Inc.

Good morning and welcome to Editas Medicine's first quarter 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, Camilla. Good morning everyone and welcome to our first quarter 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 investor 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 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, Gilmour O'Meal.

Gilmour O'Meal Thank you, Christy, and good morning, everyone. Thank you for joining us today on EDItas' first quarter earnings call. I am joined today by two other members of the EDItas executive team, Baesong Mai, our chief medical officer, and Michelle Robertson, our chief financial officer. As many of you know, in early January, we shared our strategy position EDItas as a leader in in vivo programmable gene editing and hemoglobinopathies. During the first quarter, we successfully executed this strategy driving to our goal of delivering life-changing medicines to patients with previously untreatable or undertreated diseases. We are increasing our momentum in driving our ex vivo EDIT301 program as we pursue a leadership position in hematopoietic stem cell medicines for hemoglobinopathy. As a quick recap, there are three underlying pillars to our new strategy. First, while continuing to develop EDIT301 for severe sickle cell disease and transfusion-dependent beta thalassemia, or TDT, We have sharpened our discovery focus to in vivo administer genome editing medicines. As part of that refocusing effort, we previously announced that we had divested our INK cell franchise to shoreline biosciences in January. Second, we are strengthening our discovery engine and technological capabilities. We have divided our research division into separate technology and drug discovery groups, enhancing the capabilities of each and implementing our new target selection criteria. Finally, our third strategic pillar is an increased and expanded approach to business development. In tandem, we will continue to leverage our IP portfolio to drive out licensing and partnership discussions. So, how have we executed against our new strategy in the first quarter? We have increased our investment in our Edit 301 program after reviewing promising initial Ruby Phase 1-2 study data that indicated that we have a competitive and potentially differentiated program to treat sickle cell anemia and TDT. Additionally, we are investing to develop an in vivo approach for editing hematopoietic stem cells for the treatment of sickle cell disease and TDT, leveraging the unique and differentiated approach of Edit 301 that we have already seen POC for in humans. We continue to ramp up enrollment and dosing of patients in the Ruby trial for sickle cell disease and are on track to have dosed 20 total patients by the end of 2023. We are also excited to share that the FDA recently granted orphan drug designation to EDIT31 for the treatment of sickle cell disease, and we are pleased to announce that in June, we will provide a Ruby clinical data update in an oral presentation at the European Hematology Association, or EHA, Congress, and in our company-sponsored webinar. Baesong will share further details regarding our June data readout and our enrollment progress in his remarks. On EDIT301 for TDT, we are pleased to share that we dosed the first patient in our EDITHAL Phase 1-2 trial in the first quarter, and that the patient has successfully engrafted neutrophils and platelets. Enrollment continues to progress, and we remain on track to provide initial clinical data from the EDITHAL trial by year end. Moving to in vivo, Earlier this year, our drug discovery group began lead discovery work on in vivo therapeutic targets in HSCs or hematopoietic stem cells and other tissues. As a reminder, under our new target selection criteria, we will select therapeutic targets that will allow our genome editing approach to differentiate maximally from the current standard of care for serious diseases. The target selection criteria will work to ensure targets are selected that maximize the probability of technical, regulatory, and commercial success. Our search for a new CSO to lead this drug discovery group continues to progress, and I look forward to updating you on this search and our in vivo work in the future. Turning to our intellectual property position. Since the founding of Editas, we have placed substantial importance in securing robust intellectual property protections covering our cutting-edge scientific discoveries and gene editing advancements to enable the development of novel transformative medicines for patients in need. It is important to note that we have a large portfolio of foundational US and international patents covering CRISPR-Cas9 in human therapeutics, only some of which are subject to interference proceedings. And we are confident that our IP portfolio will provide meaningful value in the future. We are the exclusive licensee of Harvard University's and Broad Institute's Cas9 patent estates. And EDItas is uniquely positioned to issue exclusive and non-exclusive licenses for Cas9 to any company seeking to use these enzymes to make human medicines, including in in vivo and ex vivo therapeutic applications. Our unique position as the exclusive licensee of this patent estate ensures that we are the party responsible for any licensing discussions as CRISPR-Cas9 products enter the market, which, given the size of the US patient market and the number of companies vying to develop CRISPR-Cas9 medicines, is a substantial position. With our newly sharpened strategic focus our world-class scientists and employees, and our keen attention to execution, we continue to build upon the momentum from our clinical readout milestones during the fourth quarter of 2022. We look forward to updating you on our progress and on the execution of our new strategy throughout the year. Now, I will turn the call over to Baesong, our Chief Medical Officer. Baesong.