Editas Medicine, Inc. (NASDAQ:EDIT) Q4 2023 Earnings Call Transcript

Gilmore O’Neill: Thanks very much. So from the in vivo pipeline point of view with regard to the how and the when, we’re excited to be on track towards POC this year for an in vivo preclinical POC. And we’re going to be able to share more and look forward to sharing more later in the year about the forum and the timing, whether it be a scientific forum or some other forum in which we would share the data. And we haven’t made a determination yet about that. And as I say, our focus is on driving towards a POC for in vivo this year. Oh, sorry. With regard to the editing tools, well, we have, I think, been very clear that we are focusing on our AsCas12a editor. And we are really focused on that, because for a number of reasons. First, it’s our proprietary enzyme that we have selected going forward, because of its high fidelity and high efficiency, because of the benefits of it using a smaller guide and the advantages for quality, et cetera, in the manufacturing.

And then finally, because we have human proof-of-concept, we have very exciting, robust editing data in human cells from AsCas12a from our reni-cel essel program. So all of those are the reasons why we’re favoring and using that editing tool. And in regards to delivery, we are using a non-viral, focusing on non-viral delivery and nanoparticles.

Jay Olson: Super helpful. Thank you so much.

Gilmore O’Neill: Thank you.

Operator: Our next question comes from Yanan Zhu with Wells Fargo. Please proceed with your question.

Yanan Zhu: Great. Thanks for taking our questions. We’re just wondering about the mid-year readout from the RUBY trial. I think you have 18 patients dosed to-date. Are you going to report data on these 18 patients plus any additional patient dosed with a certain amount of follow-up at the time of readout? And also, it seems like this is a much bigger number, compared with the last readout. So I was just wondering your confidence level of continuing to show the total hemoglobin normalization – kind of goal in this mid-year readout? Thank you.

Gilmore O’Neill: Thank you, Yanan. Before passing to Baisong on some of the details, I agree with you. Yes, this is a much bigger number of patients that we have dosed. And our confidence, as we’ve said, has increased with the accumulation of data that are continuing and repeatedly show that not only are we achieving robust fetal hemoglobin expression in excess of 40%, which is well above the – 30% threshold that natural history would tell us is relevant. But we actually also see that consistent correction of anemia normal physiologic range in all men and all women treated and followed past four months to-date. And yes, we’re excited about the mid-year disclosure. But with that, I’m going to pass it to Baisong to give you a little more detail.

Baisong Mei: Yes. Thanks, Yanan. I mean, your understanding is absolutely correct. We already dosed 18 patients and we’ll continue to dose patients in the coming months. So the data set will be 18 patients plus more that, we’re going to be dosing before the middle of the year. And as you can see, we publish the data in ASH and some patients already over a year. Then this patient will have continued to monitor for those patients longer for a period. And then we have 18 patients dosed now. And by the middle of the year, and this will have this patient, 18 patients, we’ll have three or five months more data by the time we release that. That’s why we describe as really very meaningful substantive data we will be able to share the middle of the year. As exactly you mentioned, this data set is pretty strong.

Gilmore O’Neill: Yes. And we actually, I think, Baisong, I think you said it, but it’s worth reemphasizing, we’re talking about a range of follow-ups from an efficacy point of view, between three and 18 plus months. So when you’re looking at the total patients now, we are now really building not just a data set that is robust in number, but actually robust in follow-up period time, which obviously relevant not just to our hematological and efficacy outcomes, but actually also increasing our confidence in durability.

Yanan Zhu: Great. Thanks. Looking forward to that.

Operator: Our next question comes from Luca Issi with RBC Capital. Please proceed with your question.

Luca Issi: Oh, great. Thanks so much for taking my question. Congrats on all the progress. Maybe Baisong. Any update on the grade 2 polycythemia case that was potentially related to reni-cel? I remember the poster at ASH actually noted the causality of the AEs was being investigated pending additional lab tests. So just wondering if you have any update on that one. And then maybe just quickly, any update on partnering sickle cell disease, ex-U.S.? Thanks so much.

Gilmore O’Neill: So Baisong will take that first question. And you know, with regard to partnering, I will just say that we are keen that a partner with a global footprint could help us with a global commercialization and development. We see that as an upside. But right now, our focus is on driving our sickle cell and thalassemia programs here in North America. And as I say, partnering will be something that we will look to in the future as upside. With regard to the erythrocytosis, Baisong?

Baisong Mei: Yes. Thanks. That specific patient will have a transient elevation of total hemoglobin as we reported in ASH. At the time of ASH reporting, it’s already been normal for more than six months. And that continued with the patient hematological parameter including total hemoglobin continues to stay normal. And then the investigator had a further investigation of the patient data. And we reviewed that to. And the patient, the investigator considered this event is not related to the sickle or the reni-cel treatment.