Editas Medicine, Inc.

Good morning. Welcome to Editas Medicine's fourth quarter and full year 2022 conference call. All participants are now in 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 Ron Moldaver, Investor Relations for Editas Medicine.

Thank you, Sherry. Good morning, everyone, and welcome to our fourth quarter and full year 2022 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 on 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 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, Gilmar O'Neill.

Thank you, Ron, and good morning, everyone. 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. Last year was a landmark year in EDIHAS Medicine's history as our team demonstrated two clinical proof of concepts in patients using both in vivo and ex vivo therapeutic approaches. In November, we announced data demonstrating human proof of concept for our in vivo EDIHAS 101 AAV-delivered Cas9 therapy for LCA10 and inherited retinal disease. However, due to the limited addressable population, we made the tough decision to pause patient enrollment in our brilliance trial. In December, we shared very encouraging initial data from the Ruby Phase 1-2 clinical study of our ex vivo autologous EDIT301 therapy in severe sickle cell disease. The readout provided human proof of concept, demonstrating that EDIT301 could safely drive expression of fetal hemoglobin to clinically meaningful levels and correct anemia in sickle cell disease patients. And the study continues to progress. As we move into 2023, We will build on these successes as we continue to drive towards our goal of delivering life-saving medicines to patients with previously untreatable or undertreated diseases. As many of you know, last month, we shared our strategy to position Editas as a leader in an in vivo program for gene editing. There are three underlying pillars of our strategy, which I will recap here. First, we have sharpened our discovery focus to in vivo administer genome editing medicines while continuing to develop EDIT301 for severe sickle cell disease and transfusion-dependent beta thalassemia, or PDT. We continue to support our partnered cell therapy programs, but are no longer discovering or developing standalone cell therapies, and we have divested our INK cell franchise to shoreline biosenses last month. Additionally, we terminated our AAV IRD program or platform and are seeking to divest those assets. Our sharpened discovery focus allowed us to concentrate our talent, which reduced our headcount by approximately 20% and extended our cash runway into 2025. Turning to our second strategic pillar, we are strengthening our discovery engine and technological capabilities. We have split our research division into separate technology and drug discovery groups, enhancing the capabilities of each. 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. Our goal here is to build a robust pipeline of assets that maximize the probability of technical, regulatory, and commercial success. Our new technology group under the leadership of Chief Technology Officer Bruce Eaton will focus on targeted delivery and enhancing our gene editing toolbox to enable targeted gene repair. Furthering innovation in our technology platform is a key component to achieving our goals via both internal clinical execution and external business development. Bruce is a biotech veteran who, in addition to his new role, will continue as chief business officer to spearhead our BD objectives. Within our drug discovery group, we have begun lead discovery work on in vivo therapeutic targets in hematopoietic stem cells or HSCs and other tissues. Our search for a new CSO who will head up our drug discovery group continues to progress, and I look forward to updating you on this search and our in vivo work in the future. Finally, our third strategic pillar is an increased and expanded approach to business development. Going forward, we will pursue the right combination of gene editing and targeted delivery tools through internal development and the in-licensing of complementary technologies in order to expedite our drug discovery and clinical execution objectives. In tandem, we will continue to leverage our IP portfolio to drive potential out-licensing and partnership discussions. Moving to our development plans under the new strategy, as we shared last month, we are pursuing a leadership position in HSC therapeutics for hemoglobinopathies by taking three actions. First, we have reallocated investment resources to accelerate the clinical development of EDIT301 for the treatment of sickle cell and beta thalassemia. The positive initial data from our Ruby Phase 1-2 clinical study was a key driver of our decision to invest more heavily into Edit 301. Second and third, by leveraging our unique and differentiated approach of 301, we are investing to develop milder forms of patient preconditioning, as well as develop an in vivo approach for editing hematopoietic stem cells, or HSCs, both of which should reduce the burden and improve the journey for patients who currently live with sickle cell diseases and TBD. Beyond hemoglobinopathies, our discovery and development efforts will be focused on in vivo administered genome-editing medicines in other tissues. Turning to the clinic, since we shared our strategy update last month, we have completed review of the safety data from the sentinel patients of the Ruby trial for sickle cell disease and have begun parallel dosing of additional patients. we remain on track to provide an update on the Ruby clinical data mid-year and dose 20 total patients by year-end, an ambitious but certainly attainable goal. On Edit 301 for TDT, we remain on track to dose the first patient in our Editphile Phase 1-2 trial this quarter and provide an update from this trial by year-end. Taking a step back, I am confident in our strategy, and we remain keenly focused on execution. We are building upon the momentum from our clinical readout milestones during the fourth quarter and our recently announced deal with Shoreline. We look forward to updating you on our progress and execution of our new strategy throughout the year. Now, I will turn the call over to Baesom, our Chief Medical Officer.