Editas Medicine, Inc.

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 factor section of our most recent quarterly report on Form 10-Q, which is on file with the SEC. 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, Cindy Collins. Cindy Collins Thank you, Mark.

Good afternoon, and thank you, everyone, for joining us for our corporate update call for the third quarter of 2020. In addition to Mark, I am joined by Charlie Albright, our Chief Scientific Officer and Michelle Robertson, our Chief Financial Officer. Our progress during the third quarter has laid an important foundation for the advancement of our best-in-class in vivo and ex vivo CRISPR medicine pipeline. We resumed dosing in the Brilliant Phase 1-2 trial, the first clinical trial of an in vivo CRISPR medicine, while advancing our engineered cell medicine to treat sickle cell disease and cancer towards the clinic. Starting with our in vivo programs, regaining full operating control of our ocular programs through our new agreement with AbbVie provides us with valuable flexibility as we work to strategically advance our pipeline. We have transferred the CRO contracts and the IND for the Phase 1-2 brilliance trial of EDIT-101 for LCA10 back to Editas, and we plan to finish transferring CMC activities in the fourth quarter. We are pleased to report that we have completed dosing of the first cohort in Brilliance, and enrollment remains active. However, we are experiencing delays due to the ongoing COVID-19 pandemic, which has made the logistics of screening and dosing patients more challenging particularly patients residing outside the United States. We are making every effort to accelerate enrollment in this landmark trial that holds the potential to make blind people see again. Turning to our ex vivo CRISPR cell medicines, we remain on track for an IND filing for Edit 301 in the fourth quarter and are proud to be one step closer towards bringing a potential best-in-class medicine to patients with sickle cell disease. We will present new EDIT 301 data as well as our progress in building a robust, scalable manufacturing platform for the program at the upcoming American Society of Hematology or ASH meeting in December. On the regulatory front, we were pleased to announce that we received rare pediatric disease designation from the US FDA for EDIT 301. This designation is a significant milestone that highlights the serious and life-threatening nature of sickle cell disease. In oncology, we are advancing Edit 201, our allogeneic NK cell medicine, to treat solid tumors. We will present the preclinical data at ASH, which show increased effector function of Edit 201 as compared to non-edited NK cells and we'll also show data at the upcoming Society for Immunotherapy of Cancer, or CISTI, annual meeting. Before handing the call over to Charlie for more detailed updates across our pipeline, I would like to touch upon some corporate highlights from the quarter. Our manufacturing agreements with Azure Group and Catalan provide us with capacity and flexibility as we advance our programs into the clinic. These agreements support the preclinical and clinical development of our pipeline, including Edit 101, Edit 301, and Edit 201. We are proud to have established a strong manufacturing program to support the development of our programs, particularly given the importance of having scalable, clinic-ready manufacturing in place to support the delivery of AAV and cell-based therapies. On the intellectual property front, we were pleased with the U.S. Patent and Trademark Office recent decision granting the Broad Institute's motion for priority benefit in the ongoing interference proceedings regarding certain Broad CRISPR-Cas9 patents exclusively licensed by Editas Medicine. As a result of this decision, Broad enters into the priority phase of the interference as the senior party while the opposition remains the junior party for purposes of determining which entity was the first to invent the use of CRISPR-Cas9 for gene editing in eukaryotic cells. As a reminder, the junior party carries the burden of proof in the proceedings. The proceedings remain ongoing with motions, requests, and observations expected to be filed by both parties over the next six months in preparation for another round of oral arguments. We look forward to the resolution of these proceedings and remain confident that the outcome will be a positive for the Broad Institute and, by extension, Editas Medicine. With that overview, I would now like to turn the call over to our Chief Scientific Officer, Charlie Albright, to discuss our pipeline updates.

Thanks, Cindy, and thank you for joining the call today. starting with our in vivo editing medicines. We continue to make progress with our ocular programs and are pleased to report the completion of dosing of the first cohort in the Brilliance Trial Edit 101 for LCA10. As Cindy mentioned, we've experienced slower enrollment due to the COVID-19 pandemic and remain eager to advance the trial to bring this potentially transformative medicine to patients with LCA10. Switching gears to our engineered cell medicines, an important strategic pillar at EDItos that continues to gain momentum, we continue to make progress with EDIt 301, our autologous cell medicine being developed as a potential best-in-class durable medicine for sickle cell disease. We remain on track to file an IND for EDIt 301 by year end. Further, we've identified the lead principal investigator of the trial, engaged a contract research organization to support the trial, and plan to hold an investigator meeting before year-end. As a reminder, EDIT-301 leverages our proprietary Cas12a to directly edit the beta-global locus where the mutation that causes sickle cell disease is found. Editing at this site increases fetal hemoglobin more than can be achieved by editing at the BC11a enhancer region. We believe this increased fetal hemoglobin will translate into better patient outcomes. Furthermore, our editing approach is supported by human genetics, as some people with high levels of fetal hemoglobin due to mutations in the region we are editing do not have symptoms of sickle cell disease. In contrast, editing at the BCLNA site is not supported by human genetic data. We look forward to presenting additional data on Edit 301 at the upcoming ASH annual meetings. The presentation will detail successful on-target editing of CD34 cells from healthy donors and sickle cell disease patients with our proprietary Cas12a. In these studies, we will show that editing was efficient at greater than 90% and specific as there were no measurable off-targets. Further, editing of CD34 cells from healthy donors and sickle cell patients led to robust fetal hemoglobin inductions. Red blood cells derived from editing of the sickle cell patient CD34 cells show that this increased fetal hemoglobin reduced the negative consequences of sickle hemoglobin. Finally, we will show more than 90% editing of CD34 cells from healthy donor and sickle cell disease patients with a functionally closed, semi-automated system. We know that we are editing the long-term progenitors in this experiment since we found more than 90% engraftment of the edited cells in mice. These data together support our ability to manufacture EDIT301 at scale. Taken together, these data support the advancement of EDIT301, a medicine with the potential to transform the lives of sickle cell patients. Moving next to our oncology programs, we continue to make progress with EDIT201, our edited healthy donor-derived NK cell medicine. As a reminder, EDIT201 uses our proprietary Cas12a to knock out the CISH and TGF beta genes. CISH is a negative regulator of the IL-15 pathway, a major survival pathway for NK cells. TGF-beta is a well-known inhibitor of the immune cell function in the tumor microenvironment. We look forward to presenting data at the upcoming ASH and CITC meetings. At ASH, we will present data showing that NK cells with CISH and TGF-beta knockouts are more potent than unedited cells, particularly in the presence of TGF-beta. We also show that the combination of therapeutic antibodies with Eta-201 further enhances tumor cell killing. These and other data support the continued advancement of Eta-201, a medicine with the potential to provide valuable benefit to cancer patients. We also continue to progress our engineered iPSC-derived NK cell medicines. At the upcoming ASH meeting, we will show that knockout of CISH and TGF-beta with Cas12a enhances INK potency. Specifically, double knockout clones, which were differentiated into INK cells, were more effective than control INK cells in killing tumor cells in a spheroid model. These data support the potential of our INK program as an off-the-shelf cell therapy to address a broad range of oncology indications. Further, we expect that these INK cells will avoid the challenges associated with T-cell immunotherapies, such as graft-versus-host disease, and cytokine release syndrome. We're excited by our progress, and we will continue to provide periodic updates on our INK program. Overall, we've made considerable progress across our two pillars. We're on track to file our IND for EDIT 301 this year, and we continue dosing of EDIT 101, the first-ever CRISPR in vivo medicine. We're proud of our progress and look forward to updating you in the future on our pipeline progress. With that, I'll turn the call over to our Chief Financial Officer, Michelle Robertson.