Editas Medicine, Inc.

Good morning, and welcome to Editas Medicine's third quarter 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 Christy Barnett, Corporate Communications and Investor Relations at Editas Medicine.

Thank you, Rob. Good morning, everyone, and welcome to our third 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 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 Medicine's third quarter earnings call. I'm joined today by four other members of the Editas executive team. Baesong Mai, our chief medical officer, Eric Lucera, our chief financial officer, Linda Berkley, our chief scientific officer, and Karen Dierdorf, our new chief commercial and strategy officer. We are pleased with Editas' momentum and progress in the third quarter, and I look forward to sharing these details. Before that, however, Let's take a quick step back to provide perspective. Editas' goal is to deliver life-changing medicines to patients with previously untreatable or undertreated diseases. I joined Editas in June 2022 to help realize this goal, tasked with guiding the company's evolution from a platform development company to a commercial therapeutics company. As many of you know, in January of this year, we shared Editas' vision, and strategy to position EDItas as a leader in programmable gene editing. As a reminder, three pillars underpin our strategy. First, to accelerate the clinical development of EDIT-301, our autologous ex vivo gene-edited medicine for severe sickle cell disease and transfusion-dependent beta thalassemia, and drive it towards approval and launch. Second, to 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 out-licensing our robust IP and know-how to maximize the use of CRISPR-based medicines. At the start of 2023, we outlined the following 2023 objectives. For Edit 301, to provide a clinical update from the EDIT301 Ruby trial for severe sickle cell disease, or SCD, by the end of 2023, to provide a clinical data update from EDIT301 Edithal trial for transfusion-dependent basothalassemia, or TDT, by the end of 2023, and to have dosed 20 total patients in the Ruby trial by the end of 2023. For in vivo medicine development, hire a new chief scientific officer with specific expertise aligned to our vision, and to advance the discovery of in-vivo editing of hematopoietic stem cells, or HSCs, and other tissues. And for business development, to leverage our robust IP portfolio and business development capabilities to drive value and to complement our core gene editing technology capabilities. So, how have we executed against this strategy and these objectives in the third quarter? Let's start with EDIT 301. First, on clinical data. We will present a company-sponsored webinar in tandem with a poster presentation at ASH both on December 11th, that is next month. We plan to share clinical data from 11 sickle cell patients in the Ruby trial and six beta thalassemia patients in the EDIT trial. Baesong will share more details about our presentation later on the call. Second, on enrollment. We have enrolled 27 sickle cell and eight beta thalassemia patients into our Ruby and Edifal studies, respectively, and screening continues at a good pace. Third, on dosing, we now expect to dose the 20th patient in the Ruby trial in the January 2024 timeframe due to individual patient schedules. And finally, for 2024 data disclosures, we remain on track to present a substantial clinical data set of sickle cell patients with considerable clinical follow-up in the Ruby study in the middle of 2024. Baesong will share further details regarding our December data readout and clinical progress of EDIT301 in his remarks. On the regulatory front, we are also pleased that just two weeks ago, the FDA recently granted us a Regenerative Medicine Advanced Therapy, or RMAT designation, to EDIT301 for the treatment of severe sickle cell disease. Advantages of the RMAT designation include all the benefits of the fast track and breakthrough therapy designation programs, including but not limited to intensive FDA guidance on efficient and expedited drug development, possible rolling review, and priority review of the BLA. With respect to commercial plans, as we previously shared, we made another important hire as we continue to gain momentum in pursuing a leadership position in hematopoietic stem cell medicines for hemoglobinopathy. In late September, we announced that Karen Dierdorf, a highly experienced and successful therapeutics commercial leader, has joined Editas as our new Chief Commercial and Strategy Officer. Karen has a proven ability to translate early discovery and clinical assets into robust business strategies with disciplined portfolio prioritization and value creation. Additionally, she has led multiple successful U.S. and global product launches, Karen's expertise and track record make her the ideal leader to help Editas reach this goal for patients. To further enable commercialization, as previously shared in July, we will increase our clean room capacity when we move our CMC team into the new Azure Devon facility in early 2024. With this increased capacity, we ensure our ability to scale Edit 301 manufacturing, both for clinical supply for our Ruby and Edital trials, as well as to prepare us for commercial readiness. In a step forward for the gene editing industry and patients alike, we were delighted to see the recent XSL ADCOM. The very focused review by FDA and the ADCOM confirmed our confidence in the robust nature of our own off-target assessment. The patient testimonials, in addition, were incredibly moving and powerful and demonstrate the significant need for new and transformative medicines for the treatment of sickle cell disease. Turning now to in vivo and our pipeline development, as stated earlier this year, our drug discovery group began lead discovery work on in vivo therapeutic targets in hematopoietic stem cells and other tissues. And in July, we hired Linda Berkley as our chief scientific officer to spearhead these efforts. Linda looks forward to sharing more at the appropriate time. 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 identify targets that maximize the probability of technical, regulatory, and commercial success. Now let's turn to business development. In August, we shared that we entered into an agreement with Vorbio, providing a non-exclusive license for ex vivo Cas9 gene-edited HSC therapies for the treatment and or prevention of hematological malignancies. Under this agreement, Editas received an upfront payment and will be eligible for future development, regulatory, and commercial milestone payments, as well as royalties on medicines utilizing the related intellectual property. Turning to our intellectual property position, as a reminder, Editas holds a large portfolio of foundational U.S. and international patents and is the exclusive licensee of Harvard University and the Broad Institute's past nine patent estates covering Cas9 use in developing human medicines. Only a small fraction of these patents are involved in the ongoing USPTO interference proceedings. As the exclusive licensee, we are uniquely positioned to issue exclusive and non-exclusive sub-licenses for Cas9 to any company seeking to use these enzymes to make human medicines, including in vivo and ex vivo uses. Our recently announced licensing deal with Vorbio further bolsters our confidence that our IP portfolio provides meaningful value now and in the future. To conclude my remarks, we are energized by the promising efficacy and safety data we shared in June, signaling that EDIT301 may be a clinically differentiated, one-time durable medicine that can provide life-changing clinical benefits to patients with sickle cell disease and beta thalassemia in the long term, specifically driving early and robust correction of anemia, and sustained increases in fetal hemoglobin. 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. I will now turn the call over to Bae Song, our Chief Medical Officer.