Editas Medicine, Inc.

Good morning, and welcome to Editas Medicine's second 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, Maria. Good morning, everyone, and welcome to our second 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 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 for Editas' second quarter earnings call. I am joined today by three other members of the Editas executive team, our chief medical officer, Baesong Marai, our new chief financial officer, Eric Lucera, and our new chief scientific officer, Linda Berkley. I joined Editas one year ago, tasked with guiding its evolution from platform development company to commercial therapeutics company. In January of this year, we shared Editas' strategy and a set of objectives for 2023. The strategy, just as a reminder, comprises three pillars. First, to accelerate the clinical development of Edit 301, our autologous ex vivo cell therapy for sickle cell disease and beta thalassemia, and drive toward approval and launch. Second, to strengthen our discovery organization by dividing it into an advanced technology group and a translational therapeutics discovery group, and so sharpen our discovery focus to in vivo editing therapies. And third, expand our business development activities to partner complementary technological capabilities. that can advance our in vivo pipeline development in addition to outlicensing our robust IP and know-how to maximize the use of CRISPR-based medicines. Our 2023 objectives are providing a clinical update from the EDIT301 Ruby trial by the end of 2023, providing a clinical update from EDIT301 Edithal trial for transfusion-dependent thalassemia by the end of 2023, dosing 20 total patients in our EDIT301 Ruby trial by year end, hiring a new chief scientific officer with specific expertise aligned to our vision, which we have just done, advancing discovery of in vivo editing of hematopoietic stem cells and other tissues, and leveraging our robust IP portfolio and business development to drive value and complement for gene editing technology capabilities. So how are we executed against this strategy and these objectives in the second quarter? On the leadership side, we made two important hires to drive our new strategy. Just last week, We announced that Linda Berkley, a respected scientist and highly experienced and successful therapeutics discovery leader, has joined Editas as our new chief scientific officer. Linda has an outstanding track record of inventing or contributing to the foundations of multiple approved medicines and late stage clinical candidates. We're looking forward to Linda's leadership as we build on the current in vivo editing work in hematopoietic stem cells and other tissues. Also, eight weeks ago, we announced that Eric Lucera, A highly experienced former biotech buy-side investor, chief financial officer, and strategic corporate finance executive had joined Editas as our new chief financial officer. You will hear from him later on this call. As a reminder, under our new target selection criteria, we will select therapeutic targets that allow our genome-editing approach to differentiate maximally from the current standard of care for serious diseases. The target selection criteria will work to identify targets that maximize the probability of technical, regulatory, and commercial success. Linda will tell you about these developments at a future date. We remain on track to dose 20 Ruby patients by the end of 2023, and we commenced parallel dosing of patients in our EDITHAL trial in the second quarter of this year. In June of this year, we presented clinical data from our Ruby trial at the European Hematology Association's annual scientific meeting and data from both our Ruby and EDITHAL trials at a company-sponsored webinar. We remain on track to provide another clinical trial update by year end for both trials. We will share a further update on enrollment progress in the coming months. Baesong will share further details regarding the development at 301 in his remarks, as well as recapitulate the Ruby and EDIFAL takeaways and clinical data that we provided in June. And as we have previously stated, we plan to engage with the FDA in the second half of the year. After IHA, and our company-sponsored webinar, we raised approximately $117 million in net proceeds from our follow-on issuance of common stock, extending our cash runway into the third quarter of 2025 as we continue our advancement of EDIT 301 towards BLA, prepare for commercial manufacturing of EDIT 301, and build our pipeline to transform the treatment of serious diseases. As we progress towards our goal of delivering life-changing medicines to patients, We also continue to expand our footprint to support our manufacturing and quality management for clinical supply and for preparation of commercial launch. We recently increased our clean room capacity and are moving to a new Azure Devon facility. This new facility and increased capacity will support the scaling of EDIT 301 program, manufacturing clinical supply for our Ruby and EDITF-L trials and prepare us for commercial readiness. Turning to our intellectual property position, as a reminder, EDItas holds a large portfolio of foundational US and international patents and is the exclusive licensee of Harvard University's and Broad Institute's Cas9 patent estates covering Cas9 use in making human medicines. Only a small fraction of these patents are involved in the ongoing US PTO interference proceedings. We remain confident that our IP portfolio provides meaningful value now and in the future. We are energized by the promising efficacy and safety data that we shared in June signaling that EDIT301 may be a clinically differentiated, one-time, durable medicine that can provide life-changing clinical medicine or benefits to patients with sickle cell disease and beta thalassemia long-term, specifically driving early and robust correction of anemia and sustained increases in fetal hemoglobin. With our sharpened 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. We look forward to updating on our progress in executing our new strategy throughout the year. Now, I will turn the call over to Bei-Sang, our Chief Medical Officer.