EDIT - NASDAQ

Good morning and welcome to the Editas Medicine’s Second Quarter 2024 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 Cristi Barnett, Corporate Communications and Investor Relations at Editas Medicine.

[Operator Instructions] And we will take our first question from Jack Allen with Baird. Your line is open.

Yes. Thank you, Jack. You did break up a little bit. So thanks Gilmore for elaborating there. So, yes, we are on-track to establish pre-clinical POC in-vivo for this undisclosed indication by end of year. We have not yet disclosed the CGs in which we are working, but our evaluation process is going to entail, evaluating the biodistribution to the site of interest, the efficiency of editing, the level of target modulation measured, for example, by biomarker readout and, of course, the tolerability. We will be sharing more information, the forum and the timing for announcement of that data at a future time.

Great, thank you so much for taking the question.

Thank you. We will take our next question from Samantha Semenkow with Citi. Your line is open.

Yes. Thank you, Sam. Thank you for the question. We are evaluating, LMPs with different partners because we are interested in targeting different tissue cell types for different disease areas of interest. We haven’t yet disclosed which, tissues we are doing the non clinical POC targeting in, and we will be sharing that information at a future time when we disclose the data. So in terms of the targeting, aspects that you referred to, we will share that information in the future.

Thank you. Our next question is from Eric Schmidt with Cantor Fitzgerald. Your line is open.

Thank you for the question, Eric. We have announced that in June, we have dosed more than 20 patients and we are continuing to dosing patients. So that is moving along very well. And then, we continue to consider the CATXV approval as our benchmark. So that in terms of sample size, in terms of efficacy sample size as well as observational duration of 15 to 18-months and the total sample size initial submission was 20 patients and with additional 10 patients doing the review. So this is kind of the scoop of that. And we have not shared the specifics of the time line, because we have to get a final alignment with the FDA, but we are very optimistic that, we are collecting the data from - we are dosing closing to the BLA data package we are looking for.

Thanks, Eric. We expect to have data from longer duration of follow-up for those patients we present at EHA, which means, we are going to have more than 10 patients at one year exposure and additional patients have a different exposure. And then we also at EHA, we present data for 18 patients. We have seen dosing multiple more patients, so we are going to have more patient data also. With that, we were very excited for the data end of the year because we are going to have longer duration and we are going to have a data package similar to the - So, we are looking forward to sharing data with you all.

Thank you. We will take our next question from Terence Flynn with Morgan Stanley. Your line is open.

Great. Thanks, Nia, for the question. First, we really want to say that we are encouraged by the Vertex update of the 20 patients in cell collection, which is a nice bump up from the five in Q1, and we really see that as an acceleration that we have anticipated, because we understand that there are multiple steps in the process to get a patient dosed. And we anticipate that they probably have a number of additional patients in earlier enrollment phase before cell collection, including getting payer reimbursement. We anticipate to significantly more uptake over the coming quarters as we look at both companies. We know from our own enrollment and the excitement in our trial that, there is a lot of patient and physician interest, and so that will continue as there is more exposure to our three therapies. In the U.S., we also know that payers are doing case by case approvals with few rejections. We know that, patients are able to get access and that policies will be in place over time. As a reminder, we really believe that our fast follower timing is an advantage, which gets to your question about the learnings. And that really allows us to be able to optimize our own launch plan. And another key component is that we understand the time it takes for centers to get up to speed, preparers to put policies in place. All of that, we anticipate, will come together around the time that the reni-cel might launch. We are already seeing a decrease in the amount of time for onboarding between [Bluebird] and Vertex and anticipate that, that onboarding cycle, from our own conversations and research will be significantly shorter at the time of our launch. We are really encouraged and we know, how much it takes behind the scenes, and we are just really glad to see patients moving closer to dosing. Thank you for the question.

Great. Thank you. We will take our next question from Gena Wang with Barclays. Your line is open.

Yes. Thank you, Gina, for your question. From the enrollment to dosing, it varies quite significantly among patients, and some are going to be very short, like three or four months and other can be 10 months or even longer, because some patients have BOE in the middle and doing the process before dosing of reni-cel. Between adult and adolescent, and we see now is that we have very robust enrollment and that screening process was going faster than we had for adult and the apheresis process also going very smoothly. And that is probably also related to that the adolescent patients in a better health condition compared to older adult patients.

Thanks very much, Beisong. And then, Gina, you asked a question just to clarify how we are using the indel tech to functioning or regulate gene particularly the interdiction of the regulator?

Yes. Thank you, Jamie, for the question. So our functional regulation strategy is not is different. It is not a knockdown strategy. As per your question, we would not be knocking down the repressor. If we use the reni-cel example as a paradigm. In that case, we are creating an indel to disrupt the binding site for the repressor. And that way, we are upregulating gamma globin expression. Using that as an example, we could do a similar type of thing for another target of interest. There are other ways that one can create indels to upregulate gene expression. This is the approach that we are taking that is differentiated from a knockdown approach.

Thank you. We will take our next question from Mary Kate Davis with Bank of America. Your line is open.

Yes. Thanks for the question. We believe we have a very strong foundational position with respect to the IP license that we have from Harvard, MIT and the Broad. We are very open to having conversations with a range of companies, in terms of getting a structure in place that is sort of bespoke and works for both us and them. We believe, there is a high double-digits number of programs out in development and a good number of those about half are with eight to 10 different companies. We look forward to having conversations with folks. And as you can see, we have had some pretty good success in the last few years, as evidenced by some of the licenses that we have already put in place. So very excited to have that as a potential source of non dilutive capital for our company.

Thank you. We will take our next question from Brian Cheng with JPMorgan. Your line is open.

Yes. Thanks, Brian, for the approach, the question. I think the differentiated approach is, for going after functional upregulation is really important. I think from an indication standpoint, because of the fact that, we can go after targets that others may not be able to go after using a knockdown strategy, and that is established modalities or other technologies and development, can’t go after that target, that really gives us the ability to be first-in-class. We can also envision editing strategies that would yield a better outcome than other approaches that are going after that particular indication, giving us best-in-class. So we can either have first-in-class or best-in-class opportunities here.

Thank you. We will take our next question from Joon Lee with Truett Securities. Your line is open.

Thank you. We will take our next question from Dae Gon Ha with Stifel. Your line is open.

Thank you. We will take our next question from Luca Issi with RBC Capital. Your line is open.

Yes. Luca, just following up. As we have said for many years, our intention was to look for partners outside of the U.S. And I can tell you as someone who invested in biotech for 15-years and doing business development and CFO for about 15-years. You never know where those discussions may go. But, what we can assure you is that, we are going to do the right thing in terms of what is in the best interest for getting our products to as many patients as possible and driving shareholder returns to the best level possible.

We will take our next question from Phil Nadeau with TD Cowen. Your line is open.

Thank you. We will take our next question from Jay Olson with Oppenheimer. Your line is open.

Thanks. We are very pleased with the progress of adolescent cohort and we started enrollment of this year and we already completed enrollment in the next month. So, very pleased with that. We certainly wanted to seek for broader indication of all age cohorts, and we already have alignment with FDA about the clinical trial for all the age cohorts. So that is why we are very much looking forward to that.

Thank you. We will take our next question from Yanan

Thank you. We will take our next question from Steve Seedhouse with Raymond James. Your line is open.

Yes. Thank you for the question, Nick. In clinical trial setting, we do provide fertility support, which is critical for patients, especially in this patient population, that, it is important for them to have that support to be able to go through the chemotherapy and conditioning process. I want to let Caren to emphasize the importance of that in the commercial setting.

Sorry. So MS has a section, it is the CMMI model, which is created to be able to explore and launch pilot programs to patient into different areas of need. There are numbers that have been done in oncology over the last years. And they created a specific cell and gene therapy program, of which the sickle cell disease area was highlighted and the program was initiated earlier this year or kicked off, and it is voluntary for both manufacturers and for states. So TBD on exactly who is involved in it, but the opportunity is there with a lot of funding for federal CMS through the CMMI model to be able to negotiate with manufacturers on behalf of a lot of. So it takes out some of that work in that variability. Again it is a promising program that needs to be moved forward quickly. But within it, there was a waiver on the anti kickback statute around providing the fertility preservation. So hopefully, that gives a little bit better context.

Thank you. We will take our next question from Liisa Bayko with Evercore ISI. Your line is now open.