Editas Medicine, Inc.

Good morning and welcome to the Editas Medicine first quarter 2022 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 Ron Moldaver, Investor Relations at Editas Medicine.

Thank you, Paul. Good morning, everyone, and welcome to our first quarter 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 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 and 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 Chief Executive Officer, Jim Mullin.

Thanks, Ron, and good morning, everyone. I'm joined today by several members of the Editas executive team, including Mark Sherman, our chief scientific officer, Michelle Robertson, our chief financial officer, and Charlene Stern, our general counsel. We made excellent progress this quarter and look forward to more meaningful milestones this year. In the BRILLIANCE trial for LCA10, we dosed the first pediatric patient with Edit 101. This marks the first time ever a child was dosed using in vivo CRISPR gene editing an incredible accomplishment. Given that LCA10 is one of the leading causes of inherited childhood blindness, pursuing the dosing of younger patients is potentially a critical step forward to treating this disease. And yesterday at the ARVO conference, we presented data further supporting the favorable safety and immunogenicity profile at a one-on-one. We expect to complete dosing of our pediatric mid-dose cohort in the first half of 2022 and initiate our pediatric high-dose cohort later this year. We're also planning on providing clinical updates for EDIT 101 in the second half of the year. Later today at the ARVO conference, we'll present non-human primate data with EDIT 103 for retinitis pigmentosa, demonstrating the unique design of our in vivo knockout and replace candidates to preserve retinal function That program is advancing towards IND enabling studies, which we expect to start by the end of the year. Our Edit 301 program for sickle cell disease and beta thalassemia continues to screen and enroll trial patients. For sickle cell, we've successfully edited several patients' cells ex vivo and are on track to begin dosing before the end of next month and for beta thalassemia later this year. Our cellular therapy programs are advancing. We showed impressive INK preclinical data We'll have new data on added 202 later this month at ASGCT, and we're pleased to have our longstanding partner, BMS, opt into a seventh alpha beta T cell program. The U.S. Patent and Trademark Office recently issued a favorable patent interference decision to the Broad Institute affirming our foundational CRISPR Cas9 intellectual property position Charlene's joining us today, and she'll discuss the implications of that decision in more detail in a few minutes. And finally, we're excited to announce a new leader for our organization, Gilmore O'Neill, and I'll comment on that in a few moments. But first, I'd like to briefly review our clinical programs. As mentioned, we dosed our first pediatric patients in brilliant study for Edit 101 for LCA10. It's the first time that in vivo CRISPR gene editing medicine has been administered to a child. This is a significant milestone for many young individuals living with disease, their families, as well as for the medical and scientific field. The interest and support we've received from the entire patient community has been overwhelming, and it gives us enormous sense of enthusiasm as we continue to apply our gene editing capabilities to other genetic diseases. This is the first of four planned pediatric patients in the mid-dose cohort, which we expect to complete before the second half of the year. Then, following independent data monitoring committee assessment of the safety data, we will begin dosing the pediatric high-dose cohort, which we expect to initiate later this year. To date, Edit 101 has been safe and well-tolerated in treated patients. The viral shedding data presented yesterday at the ongoing ARVO conference supported the program's favorable safety profile. The clinical data demonstrated that EDIT-101 viral shedding was transient, indicating no systemic viral persistence following the administration. There's also no correlation between EDIT-101 dose levels and the levels of viral shedding as we expected based on other ocular gene therapies, including those using AAV5. A more comprehensive clinical update on the BRILLIANCE trial will be provided in the second half of the year. This update will include safety and efficacy assessments on all patients who have had at least six months of follow-up evaluations, including at least 12 months of data on the adult mid-dose cohort and at least six months of data on the adult high-dose cohort. We anticipate these data will lay the foundation for the registration and trial event at 101. And as we've said in the past, we're exploring the most relevant and sensitive endpoints to support the registration and trial development continuing to evaluate how we can most effectively interpret trial information while always considering what is most meaningful to the patients. In addition to the trial data, we're also looking at our natural history study data to identify the most reproducible measures. We'll present some data on that study at the upcoming TIDES conference with additional findings later in the year. Moving to EDIT 301 and our ex vivo platform, The Phase 1-2 Ruby trial for sickle cell disease is enrolling study patients, and we are in track to begin dosing in the first half of 2022 with initial top-line clinical results expected by year-end. We have completed apheresis cycles in multiple patients to collect enough cells to edit and restore a healthy level of fetal hemoglobin. These patients have already had their extracted cells edited. and we're in the process of scheduling their actual dosing where we re-infuse their edited cells. This requires an extended hospital stay so that each patient can undergo the required preconditioning regimen necessary to have their edited CD34 cells successfully administered back to them. We're also preparing to initiate the Phase I-II trial of EDIT301 for the treatment of transfusion-dependent beta-falsemia, or TDT. Edit 301 recently received a rare pediatric disease designation for beta thalassemia, which it has for sickle cell disease as well. And we remain on track to dose the first TDT patient this year. We're very excited as we approach the first patient dosing Edit 301. We believe this program can be a one-time treatment for both sickle cell disease and TDT. Our unique approach recreates the same protective mutations that result in hereditary persistence of fetal hemoglobin. Patients with these protective mutations are essentially asymptomatic. We're also utilizing our Editas-engineered AS-Cas12A enzyme, one of the most specific enzymes in the gene editing field, to make these precise, complex edits. Because of the natural validation underlying the approach grounded in human genetics, We believe EDIT 301 will be a safe and durable approach to treating both of these indications. Finally, I want to mention the upcoming management change we announced a few weeks ago. Starting June 1st, Gilmore O'Neill will take over as CEO, and I will assume the role of executive chairman. Gilmore brings an incredible breadth of experience to EDITAS, including 20 years in genetic medicine clinical development, both as a physician and a scientist. He has overseen the development of numerous successful products, many addressing rare disease indications, as they made their way from early discovery to launch and now accessible to patients worldwide. As Executive Chairman, I'll be working closely with Gilmore and the rest of the executive team to drive long-term value. When the announcement was made, one frequently asked question we received was, why now? The simple answer is that Gilmore is the right person for the job and this is the right time. His creative experience of drug development, including preclinical and medical, is exactly what Editas needs today, not in a year or two, but now. We're in an excellent position with two early-stage clinical programs making good progress with several preclinical programs, and we'll continue and enhance our technology and capabilities to address other clinically significant indications. This is exactly the right moment in time to bring in Gilmore, where we can leverage his expertise to address later stage clinical trial design, prepare the next several clinical programs, and select the next clinically relevant targets to pursue. His experience and passion for creating genetic medicine will add depth and vision to our team for years to come. Now I'd like to turn the call over to Charlene to update everyone on the recent patent decision regarding the foundational IP surrounding Cas9. For some background, Charlene was one of the first people to join Editas to launch the company in 2013, and she was and continues to be instrumental in developing the company's IP strategy. I'm thrilled to have her join us this morning. Charlene.