EDIT - NASDAQ

Good morning and welcome to Editas Medicine's Second Quarter Conference Call. 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 Cristi Barnett, Corporate Communications and Investor Relations at Editas Medicine.

Thank you, Gilmore. Good morning, everyone. Let's start with the D31 RUBY trial for sickle cell disease. As Gilmore stated in his remarks, we continue to dose and enroll patients in RUBY trial. As we have previously shared, we began parallel dosing of patients in RUBY trial earlier this year and we remain on track to dose total 20 patients in UBI trial by year-end and to provide additional clinical data by. We will share further updates on enrollment progress in the coming months. As Gilmore also mentioned, we plan to engage with the FDA in the second half of the year. In the edit trial of 831 for transfusion-dependent beta thalassemia, or TDT, we continue to dose an invocation. We commenced parallel dosing in this trial in the second quarter. We remain on track to provide an additional clinical update from the additive trial by year-end. As we have done for the RUBY trial, we are also taking multiple measures to accelerate the development of 31 for TDT and have a strong [indiscernible]. We have enrolled multiple patients who have dosed or have been completed and have 334 cells additive or in the process of prices. Turning to 301 clinical data. In June, we shared promising RUBY clinical data in an oral presentation at the European Hematology Association Congress, or EHA, followed by our company-sponsored webinar where we also presented positive initial data from the first patient treated in elite trials. The RUBY data from the EHA cover safety and efficacy data from the first 4 patients treated, including 10 months data from the first patient and 6-month data from the segment patient, the data including total hemoglobin and feeding. Excitingly, the data were consistent with the clinical results we shared in last December. I would like to take a few minutes to recap some of the Rubin editor key takeaways from our presentations in June. These include the following: the 831 drives early robust correction of lime to a normal pathological range of hemoglobin in as early as 4 months of the [indiscernible] drives robust sustained increase in hemoglobin in excess of 40%. All 4 dose RUBY patients remain free of vessel-cusive events or VOE, since 301 treatment. All dose patients, 4 RUBY patients and 1 patients showed successful neutrophil engraftment within 1 month of dosing and have stopped retail transfusion. That is on was well tolerated by patients and the safety profile was consistent with myoblastic busulfan [ph] conditioning and autologous hemopoetic stem cell transplant. And the trajectory of collection of Limiar [ph] and increased expression of fetal hemoglobin was consistent across 31 treated secrete patients and better assuming patients at the same full of time points. Looking at the data in more detail; RUBY study patient 1, total hemoglobin reached normal physiological level by 5 months after 31 infusion and these normal levels persisted during the 10 months follow-up. Patient 1's fetal hemoglobin fraction increased to 45.5% 5 months after 1 treatment persisted during the 10 months follow-up. The increase of total hemoglobin and fetal hemoglobin of RUBY patient 2, 3 and 4 follow the same trajectory as patient 1. For dive, the first patient experience with A31 resembled that of 4 RUBY patients. This first dislocation achieved [indiscernible] of 34.9% or over 4 gram per deciliter in just 1.5 months after 301 treatments. We continue to believe that 31 can potentially provide robust clinical benefit to patients, potentially provide clinical differentiation in the long-term. As we have previously stated, the choice of CRISPR enzyme and the gene editing target to switch on fem expression matters. 1 uses our proprietary ASKA enzymes to added HBG1 2 promoter. ASKA [ph] increases editing efficiency and significantly reduced uptake editing when compared to other CRISPR [ph] including CAF. HBG12-editing in human CD34 port calls resulted in greater red blood cell production, normal capacity and improve red blood cell health when compared to editing of BCL11A. Based on clinical data so far, we believe that sustained normal level of total hemoglobin could be a potential point of differentiation for AD. As a reminder, a sustained normal total hemoglobin level is an important clinical outcome for patients as the correction of [indiscernible] significantly improved quarter of life and ameliorate and organ damage. As I shared last quarter, I have been visiting our RUBY [indiscernible] our clinical trial sites and continuously speaking with investigators. I appreciate the inclusiveness and support when we investigated and study size. I'm pleased with the momentum of 31 in patient recruitment, editing and dosing in both studies. I'm excited to hear from the investigators that the patients dosed with 31 already see positive changes in their lives. We look forward to sharing additional updates as the year progresses, including additional RUBY clinical trial data by year end. Now, I will turn the call over to Erick, our Chief Financial Officer, to review our financials.

At this time, we will be conducting a question-and-answer session. [Operator Instructions] Our first question comes from Terence Flynn with Morgan Stanley.

Our next question comes from Samantha Semenkow with Citi.

Our next question comes from Greg Harrison with Bank of America.

Thanks, Greg, for the question. Absolutely, we received very positive feedback from our investigators as well as patient community. From investigators, they're really to see grad to see the consistency of our results and the normalization of the total hemoglobin. And I actually asked a specific point question to one of our investigators how do you feel the normalization of [indiscernible] and with the value he will say, -- that's absolutely very important and his own patient that already told him about the energy that the patient has and the different they have and their life change in there, too. So that's [indiscernible] clinical observation and patient likewise perspective. And related to the data from patient community perspective, -- after the June presentation, our volume of patient inquiries increased 10x over that period of time after the presentation. So we're very pleased on that too. And we continue to see receiving queries not only in U.S. but also Canada and we'll help them to moving through and understand the trial and find the visibility whether they can join us enough. So very, very good.

Great to hear the progress.

Thank you, Greg.

Our next question comes from Joon Lee from Truist Securities.

Our next question comes from Gena Wang with Barclays.

Our next question comes from Dae Gon Ha with Stifel.

Two for me as well. One, with regards to Bison, your remark on total hemoglobin as being a differentiation. I guess at what point do you kind of draw the line in the sand and look at that as a differentiator from potential competitors? And then as a follow-up to that, Vertex last night announced that there will be an AdCom for XL. I guess what are you kind of thinking about in terms of point of contention or debate or discussion as we approach that? And how would you see the AsCas-12a targeting HPG promoter as potentially raising another ADCOM when you guys go down that line?

Thanks for the question. Regarding the total hemoglobin and related differentiation, we are in the current protocol, we're already actively looking to that. And as I outlined previously, we are looking for 3 category of things. One is come from hematological and other lab values. The other one and the second is the end organ function. The third would be patient report outcome and part life. And maybe a few words on the last two. We -- in the current protocol, we monitor key organ functions such as cardiovascular, hormonary and renal function; we have multiple measures to measure the function. So we believe that the total hemoglobin increase would impact the quality of life and organ health. So that's why we focus on the end organ function. And then regarding reported patient reported outcomes, it could be a very important part of that for differentiation. For example, fatigue is the main -- one of the main complaints by sickle cell patients. And that -- and already data to show that fatigue can really relate to the anemia on that, too. And similarly, for pain, that's also another the complaint that the patients have and we also have a close monitoring of those patient reported outcome. -- main other things we can share more on [ph].

Yes. Do you want to add for the initiation?

Then your second question is about AdCom. And yes, we heard the news about the AdCom which, of course, this is a novel area that FDA which is not a surprise. And we feel that will help us to learn more about how their data look like and how the expert committee is new about or additionally how the FDA feel as well. And then related to our own mechanism of action, we do not believe the target of ASK 12a caused additional concern from a mechanism of action perspective. As I mentioned before, we actually -- our targeting of HBG1 promoter is mimicking the nature mechanism of persistent the relator perineal on that. So that's kind of the nature of mutation we are targeting too. And so -- but we're looking forward to see the outcome and the information from that come from the excess sales.

Our next question comes from Phil [ph] with Cowen & Company.

Two for me as well. First, in terms of the year-end update on RUBY and EDITHAL, what is your most recent thinking about the number of patients that will be disclosed for both trials and the follow-up for the patients in the disclosures?

Yes. Thanks for the question. Yes, we are on track those total of 20 patients by year-end. We are not disclosing the specific data to be released at the year-end but we are on track to provide a clinical update for 4 studies at the year-end.

Our next question comes from Yanan

Thank you.

Our next question comes from Rick Bienkowski with Cantor Fitzgerald.

Our next question comes from Rich Law with Credit Suisse.

My congrats to both, Erick and Linda [ph] for joining the company. So the question I have is like can you provide insight to how the partner programs are progressing, so the 11 alpha beta T cell program for BMS and the NK cell with shoreline Biosciences. And when do you expect these programs to be disclosed in more details? And then I have a second follow-up question.

Our next question comes from Jack Allen with Baird.

Yes, Jeff, thanks for that question. Absolutely, we are intended to expand this study to all age groups. So we are actively working on that. And with more tile data, we are much more comfortable to go beyond the patient population.

Our next question comes from Jay Olson with Oppenheimer.

Our next question comes from Manny [ph] with Leerink Partners.

Yes. Thanks for your question, Manny. Very good question about end points and maybe I'll just step back a little bit on that. So after I joined Editas in the middle of last year. And one thing what we're doing was actually to manage the protocol, we actually changed the primary endpoint as the complete remission of the severe VOE that is consistent with other Phase III clinical trials in a similar setting. So that's kind of the primary end point expected. Then as I mentioned, there are many end points we are looking to including the end organ functions and the PROs in that too. You have absolutely very good point about possible variations and especially the PROs in there, too. So we're looking into that direction that will be -- we have more data on that and method understanding. But we feel that this is an area that we have publications in the myself [ph]. We will have more information about the X and other data and will allow us to have an understanding of where we stand for those end points.

Great. I guess one quick follow-up. You talked a little bit about how to understand where you guys are on those endpoints. Do you have any average components to in the literature or based on experience in publicly disclosed data from other companies or your own around what duration of follow-up we would need to be able to compare across trial statistical caveats notwithstanding and kind of provide clear evidence of where you guys are versus where your competitors are? Like how long follow-up would we need to clearly differentiate on pain, on fatigue, on quality of life?

Yes, very good question. That's something we are looking into that. First of all, we say there are some information already published, for example, in the allogeneic transplant perspective for scarce disease, their publication in multiple dependent on that to see how patients improve after the moves stem cell transplant for those patients. And then in terms of specific endpoints, some will take longer and the other will take shorter time. For example, we already see the [indiscernible] increase and ratio of time period. So that's kind of probably taking shorter time wise. And so far for some point of life, it may take 6 months or 12 months ago and for the analog damage and that's actually a space that we'll continue to learn. They are actually publication, for example, to say from central nervous system and after the allogeneic transplant, they see improvement hydrodymic improvement in blood vessel in the brain. So there's a lot of studies in this space. But again, we are steering a new territory, we are looking into that. But we are confident that we will see something over the period of time to see some differentiation.

Our next question comes from Luca Issi with RBC.

Thanks so much.

Our next question comes from Liisa Bayko with Evercore ISI.

I just had -- I wanted to drill down a little bit more on the gentler conditioning regimen program. So what are you working on in terms of approaching that? What do you think are the most I guess, promising approaches out there. What is your thinking on when something like that could come to market? And I guess if someone else developed something like that, is that something you think you could fold into your program? And how do you think about sort of owning something like that versus someone else and your access to it?

Our next question comes from Brian Cheng with JPMorgan.

Our next question is from Steve Seedhouse with Raymond James.