Editas Medicine, Inc.

Good morning, and welcome to Edisys Medicine's third 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 Mulder, Justice Relations at Edisys Medicine. Please go ahead, sir.

Thank you, Maria. Good morning, everyone, and welcome to our third 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 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, Gilmer O'Neill.

Thanks very much, Ron, and good morning, everyone. I'm joined today by several members of the Editas executive team, including Baesong Mai, our chief medical officer, Mark Sherman, our chief scientific officer, and Michelle Robertson, our chief financial officer. I am pleased with the progress our team has made this quarter. We have continued to build on our company's foundational technology as we transform from a platform company into a clinical stage therapeutics company and focus on execution. We continue to evaluate ways to leverage our gene editing expertise for developing new therapeutics, and we will provide an update on this evaluation in the coming months. Operationally, with our focus on clinical advancement, execution is a top priority going forward. With that, I would like to provide some recent highlights on our clinical pipeline programs. First, edit 301 for sickle cell disease and transfusion-dependent beta thalassemia. EDIT301 utilizes a unique mechanism of action that edits the promoter of the gamma-globin gene to disrupt binding of the BCL11A suppressor. This is designed to provide high and durable levels of fetal hemoglobin in patients with severe sickle cell disease and TDT, thereby resulting in reduced red blood cell sickling in sickle cell sufferers and reduction in anemia in TDT patients. I would also note that our EDIT301 program uses our proprietary AS-Cas12A engineered nuclease, which our preclinical data suggests results in higher fidelity and higher efficiency editing than Cas9. As a reminder, the initial patient dosing with EDIT301 represents the first time that an autologous ex vivo drug product was edited using our AS-Cas12A engineered nuclease. In the RUBI Phase 1-2 trial evaluating EDIT-301 to treat sickle cell disease, we have dosed the second patient and remain on track to release initial data from the RUBI trial by year end. These data would include efficacy data from the first treated sickle cell disease patient, as well as safety data from the first two treated patients. In the EDIT-VAL Phase 1-2 study for transfusion-dependent beta thalassemia, We have completed apheresis and CD34 positive cell editing on the first patient and are currently scheduling dosing. Let us now turn to Edit 101 for LCA10, which is a devastating inherited retinal dystrophy caused by autosomal recessive CEP290 mutations that cause early severe visual impairment or blindness, and talk about the Phase 1-2 Brilliant Study. The Brilliant Study is designed to achieve several objectives. to determine the safety of delivering EDIT-101 to retinal photoreceptors, to identify a subpopulation of LCA10 patients characterized by baseline molecular, clinical, or physiological parameters who are most likely to benefit from therapy, and to identify a dose that optimizes the benefit-risk balance of EDIT-101 and to identify the optimal endpoints to consider for a registration study. An update on the BRILLIANCE trial will be provided this month through a press release and company-sponsored webinar. That readout will include safety on all-dosed adult and pediatric patients and efficacy on the adult mid- and high-dose cohorts. Data from our one-year natural history study of 26 LCA10 patients with mutations in the CEP290 gene will be used to contextualize the brilliance data. In order to move forward to a registrational study, we would need to see a meaningful treatment benefit for a commercially viable patient segment. Baesong will provide further details in his remarks. Beyond the BRINIANS trial, on the safety side, the EDER1-1 program lays the foundation for subsequent potential drug development programs that utilize our AV-based in vivo platform in inherited retinal diseases. Thus far, we have been able to demonstrate safety and tolerability from EDER1-1, reinforcing the use of AV-delivered CRISPR-based retinal therapeutics. Mark will provide an update on our preclinical pipeline, as well as a summary of data recently presented at scientific meetings. Let me now turn to our IP portfolio, which includes patents exclusively licensed from the Broad Institute and Harvard University that cover Cas9 for use in human therapeutics in the U.S. With our exclusive license, the granting of a sub-license on either an exclusive or non-exclusive basis is at our discretion. As you know, earlier this year, the Broad Institute prevailed for the third time, twice with the PTAP and once at Federal Circuit, against parties collectively known as CBC. As anticipated, the CBC appealed the most recent PTAB decision, and the Federal Circuit will review the ruling to determine whether the law was properly applied. The court will not hear new evidence, and we expect the court will deliver its decision in mid to late 2023. We remain confident that the broad will once again prevail. An appellate court decision in the Broad's favor would reaffirm Editas' position as the exclusive licensor of the Cas9 therapeutic patents in the U.S. Thus, all companies that are developing a product utilizing Cas9 and that plan to commercialize that product in the U.S. will need a license from Editas. It is important, in addition, to remember two things about Edit 301 on the IP front. First, it uses our proprietary AS-Cas12A nuclease, which is not the subject of any IP disputes. And therefore, Edit 301 will not need a CAS 9 license for commercialization. We believe that our strong IP position relates to CAS 9 human therapeutics in the U.S. has the potential to be a significant value driver for our company, with numerous competitor products in development using CAS 9, including several in late-stage clinical development. We look forward to providing additional updates. I will now turn the call over to Baithong, our chief medical officer, to review the details of our clinical programs. Thank you, Gilmore.