Editas Medicine, Inc. (NASDAQ:EDIT) Q4 2022 Earnings Call Transcript

Phil Nadeau: Great. And then two follow-up questions in terms of the prior question, the mild preconditioning, do you have a sense when Editas could move that into the clinic when you could begin testing a milder preconditioning regimen? And then second, in terms of business development, you mentioned you were looking to bring complementary technologies, in-house. Could you give us some sense of what complementary technologies you think you need? Thank you.

Gilmore O’Neill: Yes, thanks very much, Phil. With regard to the milder preconditioning, we will actually talk more about that in the future at an appropriate time when we have some more details that we feel able to share with you. With regard to the complementary technologies, just in general, we are looking across the two elements of advanced technology that will be important to driving our in-vivo focus, and those around targeted delivery and obviously additional targeted editing approaches. And again, we will be able to share more details about that in the future.

Phil Nadeau: Perfect. Thanks for taking our questions.

Gilmore O’Neill: Thanks very much, Phil.

Operator: Our next question is from Rick Bienkowski with Cantor Fitzgerald. Please proceed.

Rick Bienkowski: Hi everyone. Good morning. Thanks for taking the questions and congrats on all the progress here. So my first question is on the in-vivo pipeline. I know that the first two target indications are currently undisclosed, but it looks like the initial discovery efforts are going towards the in-vivo editing of stem cells. Could you highlight the reasons why you chose this as the initial area of focus and why it could be advantageous to target these cells rather than an organ like the liver where methods of delivering gene editors are more established? And then for my second question, for the goal of dosing 20 patients by year-end in a sickle cell trial. Are there any important limitations in manufacturing capacity or bed available that would limit the number of patients that you can dose in parallel and I guess what I’m really trying to understand here is if patient dosing is likely to be evenly distributed over the year, or if it could be weighted more towards the back half of the year?

Gilmore O’Neill: Thanks very much Rick for those questions. Let me turn to the in-vivo pipeline question, while we did talk about in-vivo HSCs, indeed we are looking at other tissues, which we have not disclosed. The reason that we actually talked about HSCs is that our several one of which is that it aligns with a key focus of our active pipeline. It builds on the success of our EDIT-301 and essentially we have somewhat simplified the calculus by having a human validated enzyme or a factor enzyme in AsCas12a and a humanly validated target in the HPPG 12 promoter, which enables us to really focus largely or predominantly on the target delivery. I will say, however, that we are actually are looking at other tissues but we haven’t disclosed those yet.

Turning to your second questions with regard to limitations, we have actually substantially invested and reallocated capital with our new strategy to CMC and do not have limitations there to both editing and supporting parallel dosing. But indeed, Baisong could probably tell you more about the enthusiasm in the community following our data disclosure and our plans for the year.

Baisong Mei: Yes, sure. Rick, yes, that is a very good question. So, as Gilmore mentioned, we really beefing up our CMC capacity as we discussed before. We actually have a strong CMC team internally be able to handle all those – handle the internal CMC manufacturing capacities and working with partners. In terms of the – we have patient recruitment, we really see a strong momentum over the last several years. So we are very pleased to see the number of patients being enrolled, in screening as well as the prospective patients we have in there. So, we are very confident we will be able to achieve the goal 20 patient by year-end.