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Q4 2023 Editas Medicine Inc Earnings Call

Participants

Cristi Barnett; Investor Relations; Editas Medicine Inc

Gilmore O'Neill; CEO, President; Editas Medicine Inc

Baisong Mei; Senior Vice President, Chief Medical Officer; Editas Medicine Inc

Erick Lucera; CFO; Editas Medicine Inc

Caren Deardorf; Chief Commercial and Strategy Officer, EVP; Editas Medicine Inc

Joon Lee; Analyst; Truist Securities, Inc.

Samantha Semenkow; Analyst; Citigroup Inc.

Brian Chan; Analyst; JPMorgan Chase & Co

Greg Harrison; Analyst; Bank of America

Mani Foroohar; Analyst; Leerink Partners LLC

Gena Wang; Analyst; Barclays Bank PLC

Dae Gon Ha; Analyst; Stifel, Nicolaus & Company, Incorporated

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Philip Nadeau; Analyst; TD Cowen

Jay Olson; Analyst; Oppenheimer & Co. Inc.

Yanan Zhu; Analyst; Wells Fargo Securities, LLC

Luca Issi; Analyst; RBC Capital Markets

Timur Ivannikov; Analyst; Raymond James & Associates, Inc.

Jack Allen; Analyst; Robert W. Baird & Co. Incorporated

Presentation

Operator

Good morning and welcome to Editas Medicine's fourth quarter and full year 2023 conference call. (Operator Instructions) 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 at Corporate Communications and Investor Relations at Enel.

Cristi Barnett

Thank you, Maria, and good morning, everyone, and welcome to our fourth quarter and full year 2023 conference call. Earlier this morning, we issued a press release providing our financial results and recent corporate updates. A replay of today's call will be available in the Investors section of our website, approximately two hours after 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 the 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 Factors section of our most recent annual report on Form 10-K, which is on file with the SEC as updated by our subsequent filings. 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 CEO, Gilmore O'Neill.

Gilmore O'Neill

Thanks, Christy, and good morning, everyone. Thank you for joining us today on any asset fourth quarter and full year 2023 earnings call. I am joined today by four other members of the other tests, executive team, our Chief Medical Officer, Baisong Mei; our Chief Financial Officer, Erick Lucera; our science, Chief Scientific Officer, Linda C. Burkly; and our Chief Commercial and Strategy Officer, Caren Deardorf.
We are pleased with Editas has momentum and progress in the fourth quarter and all of 2023 in early 2023, we shared our vision on the three pillars of our strategy to position Editas as a leader in in vivo program for gene editing and hemoglobinopathy. The first of these pillars to Dr. Rennie sell, formerly known as editorial one toward BLA and commercialization.
Second, to strengthen, reorganized and focused our discovery organization to build an in vivo editing pipeline from the third to increase business development activities with a particular focus on monetizing our very strong IP.
So how did we do last year? While we achieved a loss? First, we accelerate the clinical development of many cell, exceeding our enrollment goal of 20 patients and sharing clinical updates from our Ruby and edited studies in June and in December of 2023 and those accumulating data have strengthened our belief that Renaissance is a competitive potential medicine with a differentiated profile characterized by correction of anemia to normal physiologic ranges of hemoglobin.
Second, we strengthened our in vivo discovery capabilities and organization, and hired a new Chief Scientific Officer, Linda Barkley, who brings three decades of experience in successfully inventing, developing and moving new human medicines forward And third, we increased our business development activities and monetize our IP, leveraging our robust IP portfolio. A quick example was our granting Virtex-E non exclusive license for our cash nine IP in a focused way to enable the XL launch.
Finally, we strengthened our senior leadership team with people who have a proven track record in bringing new medicines through development to approval and commercialization. So how we executed against the strategy and these objectives will let's start with myself. First on enrollment, we have now enrolled 40 sickle cell and nine beta-thalassemia patients in our Ruby and edit out studies, respectively, and enrollment continues at a good pace.
Second, on dosing, we have dosed 18 will be patients in 7,000 patients, and we have multiple patients scheduled for dosing in the coming months. Patient screening and demand in both studies continue to remain robust. Third, on clinical data, we remain on track to present his extensive clinical data dataset of sickle cell patients with considerable clinical follow-up in the Ruby study in the middle of 2024, with a further update by year end 2024.
On the regulatory front, we have engaged with the FDA regarding the Ruby sickle cell trial. The FDA agrees that Ruby is a single Phase one two three study and have aligned with us on the study side, our discussions with the FDA will continue as will be at about progress and will be enhanced by our RMS designation for severe sickle cell disease based on, we'll share further details regarding the development of Randy said in his remarks as well as recap the Ruby and any final takeaways and clinical data that we provided in December and share more information on the adolescent cohort.
Now let's turn to in-vivo and our pipeline development, where we strengthened our in vivo discovery capabilities at 2023 and began the discovery work on in vivo therapeutic targets in hematopoietic stem cells and other tissues. As we announced earlier this year, we aim to establish in-vivo preclinical proof-of-concept for an undisclosed indication this year.
Linda and her team are leveraging key capabilities that we have in-house and shoot forward to sharing more at a future date. Regarding our hemoglobinopathy focus, after a thorough evaluation of the development landscape, we have decided not to pursue internal development of a milder conditioning regime.
We believe stand-alone monitor conditioning regiments would be widely available once FDA approved and therefore, we have determined our research, clinical development and regulatory investments in terms of open up, these can be better deployed for our continued development of in vivo HSC methods.
Turning to assist about in the fourth quarter, we announced a license agreement with Vertex Pharmaceuticals added test provided protects a nonexclusive license for Editas Medicine's cast mine gene-editing technology for ex vivo gene editing medicines targeting BCMA gene in the fields of sickle cell disease and beta-thalassemia, including Vertex's cost savings and the upfront and contingent payments pursuant to this agreement, extended our cash runway into 2026.
This and other agreements, the strength of our patents and the number of companies developing crisper Cas nine medicine reaffirm our confidence that our IP portfolio of foundational US and international patents covering cash, nine used in human medicine are a source of meaningful value. So one of our objectives for 2024, Randy, so we will provide a clinical update from the Ruby trial for severe sickle cell disease and the Edipower trial for transfusion-dependent beta-thalassemia in mid 2024 and by year end 2024.
We will complete the adult cohort enrollment and initiate the adolescent cohort in Ruby, which we've already initiated and continue enrollment and ourselves for our in vivo pipeline, we will establish in vivo preclinical proof of concept for an undisclosed indication.
And for BD, we will leverage our robust IP portfolio and business development to drive value and complement core gene-editing technology capabilities. We are energized by our progress and execution in 2023. With our sharpened strategic focus, our world-class scientists and employees and our keen drive and execution, we continue to build momentum to progress our strategy to deliver differentiated medicines to patients with serious genetic diseases.
Now I will turn the call over to Baisong, our Chief Medical Officer.

Baisong Mei

Thank you, Kim, and good morning, everyone. Let's talk about the Renaissance, which is on the clinical development of severe sickle cell disease and transfusion-dependent beta-thalassemia. As of today, it will be trial for sickle cell disease. We have enrolled 40 patients and dosed 18 patients. We have multiple patients scheduled for dosing in the coming months. We're also pleased to announce that we have initiated adolescent cohort in the Ruby study which is well above our 2024 objectives.
The interest and demand a high and adolescent patient have already started screening in the AD field trial for transfusion dependent. And Beth, you've seen year to date, we have enrolled nine patients and dosed seven patients. As I shared earlier, I continue to visit our Ruby and Eddy failed clinical trial sites and continuously speak with the investigators, I appreciate the enthusiasm and support from the investigators and study sites.
I'm pleased with the momentum of Renevia in patient recruitment, freezes, editing and dosing in both studies I'm excited to hear from the investigators that patient does with Renaissance. I've already seen positive changes in our lives.
As Gil mentioned, we have aligned with FDA. There will be clinical trial is now considered a Phase one two three trial for FI. We've also aligned with the FDA on the study design and endpoints, and the FDA had agreed to our activation of adolescent cohort. We look forward to future discussion with FDA and continued calibration.
Turning to clinical data. In December 2023, we shared safety and efficacy data from 17 patient, 11 Ruby patients, 6 [Editas] patients. Once again, the data confirmed observation from our prior clinical readouts, including benefit of driving early and robust correction of anemia to a normal physiological range of total hemoglobin in sickle cell patients. When I say job robust and sustained increase in fetal hemoglobin in excess of 40%.
All will be sickle cell patients have remained free of vessel crews, events falling rent if they are treated rent-to-sales, treat sickle cell and benefit, assuming a patient have shown successful engraftment have stopped red blood cell transfusion and the safety profile of benefit observed to date is consistent with yourself and Bob electric conditioning and autologous hematopoietic stem cell transplant.
In addition, the trajectory of the correction of anemia and expression of fetal hemoglobin is consistent across Renaissance treated sickle cell disease patient and beta-thalassemia patients at the same follow-up time points. These data reinforce our belief that we have a competitive product and the product potentially differentiated from other treatments with rapid correction of meaning.
Thanks to the deliberate choice and our discovery group has made early in the progress as we have previously stated the choice of crisper enzyme and the parliament to edit for increase the hemoglobin fetal hemoglobin expression matters, beneficial use of our proprietary Asia to our enzyme to added HBG when to promote at the ASCA. 12 A. increases efficiency of editing and a significantly reduced off target at eight when compared to other crisper nucleus, including test.
Now anything HBG. one to promote or a human CD34 positive cells without a greater red blood cell protection, normal curvature for capacity and improved red blood cell health. We'll come back to editing of BCL11A. We look for differentiation in three categories of outcome in clinical trials, hematological parameter and organ function and patient reported outcome of quality of life.
Based on the clinical data thus far, we believe that sustained normal level of total hemoglobin could be a potential point of differentiation for Renovis. As a reminder, a sustained normal to hemoglobin level is an important clinical outcome for patients and the correction of anemia can significantly improve quality of life and ameliorate and organ damage. We look forward to sharing additional updates, including Ruby and Edison clinical trial data with more patients and longer follow-up period in midyear and additional data by year end.
Now I turn the call over to Erick, our Chief Financial Officer.

Erick Lucera

Thank you based on and good morning, everyone. I'm happy to be speaking with you, and I'd like to refer you to our press release issued earlier today for a summary of financial results for the fourth quarter and full year 2023. And I'll take this opportunity to briefly review a few items for the fourth quarter. Our cash, cash equivalents and marketable securities as of December 31 were $427 million compared to $446 million as of September 30, 2023.
We expect our existing cash, cash equivalents and marketable securities, together with the near term annual license fees and contingent upfront payment payable under our license agreement with Vertex to fund our operating expenses and capital expenditures into 2026. Revenue for the fourth quarter of 2023 was $60 million, which primarily relates to revenue recognized under our license agreement with Vertex which, as Gilmore referenced earlier on this call, we announced in December of 2023.
R & D expenses this quarter increased by $18 million to $70 million from the fourth quarter of 2022. The increase reflects additional sublicense expenses offset by the decrease in R&D spend resulting from our reprioritization and targeted focus on our red cell program. G&A expenses for the fourth quarter of 2023 were $14 million, which decreased from $18 million for the fourth quarter of 2022.
The decrease in expense is primarily attributable to reduced patent and legal costs. Overall, Editas remains in a strong financial position, bolstered by our sharpened its discovery focus, June capital raise and our recent out-licensing deals. Our cash runway into 2026 provides ample resources to support our continued progress in the Ruby and Eddie Bauer clinical trials that went to sell, continued commercial manufacturing preparation and the advancement of our discovery and research efforts.
With that, I'll hand the call back to Gilmore.

Gilmore O'Neill

Thank you, Eric. We are very proud of our progress in 2023 and look forward to accelerating the momentum into 2024 as we continue to evolve from the Verizon states technology platform company into a commercial stage financing company. We look forward to continuing our transformation and sharing our progress with you.
As a reminder, our 2024 strategic objectives improved for any cell who will provide a clinical update from the result will be trials for severe sickle cell disease and the Edipower trials for transfusion dependent beta-thalassemia. In mid-2024 and year-end 2024, we will complete adult cohort enrollment and we already initiated the adolescent quarter, and we will continue enrollment in Edipower for our in vivo pipeline.
We will establish in-vivo preclinical proof of concept for an undisclosed indication and for PD., we will leverage our robust IP portfolio and business development to drive value and complement core gene editing technology capabilities. As always, it must be said that we could not achieve our objectives without the support of our patients, caregivers, investigators, employees, corporate partners, and you thanks very much for your interest in Editas, and we're happy to answer questions.

Question and Answer Session

Operator

(Operator Instructions) Joon Lee, Truist Securities.

Joon Lee

Hi, good morning and congrats on that quarter. I'm sorry for my voice. And my question is that could you please elaborate on the hemolysis markers that you are tracking and tell us how they will relate to patient reported outcomes such as quality of life.

Gilmore O'Neill

Thank you and thanks very much, Joon. I hope that your voice gets better on. I'm going to pass that question over to Basil.

Baisong Mei

Yes, thanks for the question. Joon so far this molecule, Marco, we look into multiple that Micrus fall into any for analysis, including reticulocytes, LDH, bilirubin, among others. And for the on patient reported outcome, we use several instrument and to measure that clinical outcome and quality of life and lead related to make the channel a our PRO instrument as well as sickle cell specific instrument.

Joon Lee

Thank you.

Operator

Samantha Semenkow, Citi.

Samantha Semenkow

Hi, good morning. Thanks very much for taking the question. And Peter, I just any additional insights into your FDA conversation, as you outlined on the Ruby trial, specifically in terms of the number of patients you'll need and the amount of follow-up you'll need to file potential BLA.

Gilmore O'Neill

And I'm going to have Baisong on address that question and thank you for the question.

Baisong Mei

So we aligned with the FDA about the Ruby trial and to start to be a Phase one two three trial to support a BLA, including endpoint sample size and study design. And we are doing we are continue to have engagement with the FDA about the FDA with FDA about the data package and the follow-up. And so we'll have a have further discussion with FDA.

Samantha Semenkow

Got it. Thank you.

Operator

Brian Chan, JP Morgan.

Brian Chan

Yes, hey, guys. Thanks for taking our questions this morning. Can you just kind of give us a sense of your what does the Phase one two three destination for Ruby really mean from a time line perspective and on the potential differentiation, I think based on your talk about investigators feedback so far, I'm curious if you can also talk about just a feedback that you've been hearing from investigators, are they seeing potential differentiation in this early on? Thank you.

Erick Lucera

Thanks very much, Brian. So I think there were three parts to your question, what is the phase one, two, three and its impact on sort of the DLA pass on loss? Was the investigator feedback on differentiation and where they're seeing signs, Bob or things that they might be seeing in patients at this point. And what I'll do is just address the first part and then ask Basab to follow up on the other two.
So with regard to Phase one, two three, I think the key point here is that it is a single we've agreed that there's a single Phase one two three study. And we have important agreements on what the outcomes are and the size of the study. And what that basically means is that we remain on track and are confident more confident about being on track two to a BLA.
I think it's worth calling out that at Vertex, a study was also the study that was used for their BLA application was designated a Phase one two three before prior to AT THAT THE LA. So I hope that actually helps from the point of view of our path to BLA and I think just mentioning Bertek, it was just worth calling out that we sort of had a benchmark based on the BLA filing from last year with regard to the size of the filing that was originally used for that approval based on?

Baisong Mei

Thanks. Brian, on the for the differentiation and the investigator feedback wise. And so we actually have quite a bit engagement with it with the investigator and from their all their own observation of their patient as well as I see the data, they are very pleased to see the correction of anemia.
And as a hematologist that very much I appreciate that the level of total hemoglobin be able to correct the anemia and they see their patients is less fatigue and they have more energy and they just they have direct observation. And then also they have put us that tubing level as published also impacted the end organ function. So those are the direction that we're often looking for.

Erick Lucera

And one of the thing I'd just like to quote, just with regard to the Phase one two three, I think the important thing is is that the Ruby study has been has we've agreed with the FDA. It's converted from a Phase one to a Phase one two three, which allows that because it's a single study a seamless transition it to that study to support BLA. Yes, that's helpful.

Baisong Mei

Just at Onkyo, most point, what Gilead means, it's also to say we'd be able to use all the patient data from the study to support the DOL.

Brian Chan

Great. Thanks, guys.

Operator

Greg Harrison, Bank of America.

Greg Harrison

Hey, good morning, and thanks for taking the question now that there is a gene editing treatment approved in sickle cell. What are your latest thoughts on how Renaissance would fit in the space and what have you learned from the early launch by the competitor.

Gilmore O'Neill

Thanks very much, Greg. I'm going to ask Karen to address that question.

Caren Deardorf

Yes, Craig, thanks for your question. What we've been hearing from the various stakeholders in this space is a lot of interest and some really good initial momentum. I think we're also hearing that across all of the groups that you're taking to your patient families, your centers, which are transplant, maybe gene therapy centers, your qualified centers and as well as your peers.
There's just a lot of work that needs to happen and I think you all are picking up on that, but it's starting and it's happening. So I think it's the balance of saying there's tremendous interest even from the government in the CMMI. pilot that CMS has kicked off. So that's the way we see it as is very encouraging.
It's going to take time and we really believe that the fast follower of Renaissance is going to be timed very well, gives us time to be able to continue to collect data that is meaningful, differentiated to understand where centers may be struggling and where else we can optimize our vein-to-vein process to deliver a product that's differentiated, not just in efficacy and safety, perhaps, but also in our operational aspects.
So we see the market developing in a very robust way. We just think it's going to take a little bit of time. So we're we're very pleased with initial interest. Certainly the ongoing interest in our clinical studies and look forward to talking more about it.

Greg Harrison

That's helpful. Thanks so much.

Operator

Mani Foroohar, Leerink.

Mani Foroohar

And then taking the question on, can you talk a little bit about improvements in vein-to-vein time as an incremental source of differentiation and independent clinical data? Can you walk through how you guys think of the timing on which we might see that show up in terms of CapEx investment in terms of competition, et cetera, like where we're just seeing data points you could de-risk that part of your advantage in the eyes of investors?

Baisong Mei

Yes. Thanks for the question. This is based on. Thanks. Thank you, Manny. And we are in the clinical trial stage, why we're already trying to optimize all the process from bank to bank, as you mentioned in there, too. So we amend our protocol and work with our expert in a free seats, for example, and to help with the size and on the in the recent cycle and help us size the prepare the patients and provide support. So that's kind of the experience we gather now?
Well, how far while future mark a commercial launch, I'll pass that to Karen on that.

Caren Deardorf

Yes. And just to add, as we continue to be very engaged in our clinical sites and expanding our outreach to other centers. We'll have the opportunity to really understand how the process is working, how we make the introduction of a product like run itself as seamless as possible, do the advanced work fit into their existing processes. But we're always looking at opportunities across just again the efficiency, the timing and will certainly be able to talk in a more as we get closer.

Mani Foroohar

Great, that's helpful.

Operator

Terence Flynn, Morgan Stanley.

Great. Thank you team. This [Max Goran] on for Terence Flynn. Can you provide an update on the Crystal Aircast nine appeal case and whether you expect an oral argument in the first half of 2024.

Gilmore O'Neill

Thank you and thanks very much, Max. On we have the app Court of Appeals Federal Circuit has yet to schedule that oral hearing on, which should be sometime sometime this year. And once we have that scheduling, we can update you on that and Great.
Thank you.

Operator

Gena Wang, Barclays.

Gena Wang

Thank you for taking my questions. So if I heard you correctly you more you mentioned the FD. and R. based on. I think both of you maybe mentioned a single study. If you agree on the single study also number of a patient, I'm not sure if they agree on the duration of the study will be trial.
So wondering if we think about the crisper trial, Vertex trial, it's about 45 to 50 patients. Is that online? Was that numbers? And what is the duration? The FDA requires And a related question is how many active sites you have now and what is your goal? Total sites for the pivotal?

Gilmore O'Neill

Sorry, and thanks very much, Gina, for your question. Before I let me just address a couple of things from point of view of the study design. I think first of all, the benchmark that was set by the BLA with Vertex's approval by the FDA for extra sale is actually very good benchmark and what we against which we are operating.
And that has certainly in far informed our discussions with the FDA and we were actually very pleased with the discussions with the FDA, and we believe that it's actually really puts us on a track that lines up with the benchmark. And as a result, when we actually talk about having enrolled 40 patients who have initiated the adolescent enrollment and so on.
We believe we're actually in a very good track on a four to a BLA with regard to the number of active sites, I do want to clarify something that we're not talking about a separate study, essentially the Ruby study, which is ongoing with its sites activated is the Phase one two three study that will be used for BLA, and that is the agreement that we have with the agency.

Gena Wang

Okay. And sorry, the reason I'm asking more thinking about in the future commercial perspective, if you have already established the active sites for your clinical trial, then it's very easy to transition this to commercial one drug in the future.

Gilmore O'Neill

Yes, I understand that, and I appreciate that clarification on and certainly agree with that principle. I think, Karen, if you want to add.

Caren Deardorf

No new, no. I mean, I know, we've already we had already expanded the number of sites on previously to get to a number that would support the full study, right? They punch and so that that was a very thoughtful approach to ensure that we had a good strong number of sites with geographic coverage.

Baisong Mei

Yes, maybe a competitive launch. You know that we already shared we have activated over 20 sites and to so we are with that over 20 sites. We already enrolled in 40 patients. And we already, we would know size for adolescent cohort that there will you have the overlap between adult and adolescent from the same study size and we also are activating a few more sites that specifically for pediatric patients.

Gena Wang

Thank you very much.

Operator

Dae Gon Ha, Stifel.

Dae Gon Ha

Rodrigo, thanks for taking our questions of. I apologize, it's actually a two-part question, but just to clarify on the Renaissance progress in Ruby, if I heard you correct, 18 dosed, I thought the prior conversation was 20 dose by January. So can you talk about sort of the dropouts there. What was the reason behind that?
And then another clarification on the FDA side. When you talk about differentiation, have you guys actually engage them on the angle you're taking on the differentiation, whether total hemoglobin or end organ function? How are they perceiving that in terms of the conversation? Thanks so much.

Gilmore O'Neill

Thanks very much, Jake. On first of all, I think we're actually very happy with where we are with our dosing 18 patients dosed arm. And that really has us on track and with that dosing pace to get us on track for a presentation of substantial data set in the middle of the year. And actually with regard to our driving towards BLA at we have no, we have not had dropouts so just to be sure that there's no confusion about that.
And I think the final thing is that, you know, based on your myself with our clinical experience and particularly when you're dealing with a complex therapeutic that you're going to get some waves, you know, ups and downs and waves of not just enrollment and dosing, particularly around scheduling around holiday periods. Et cetera.
So as baseline also said, we have and many more patients scheduled for dosing in the coming months. And as I say, remain on track for a substantial data set in the middle of the year with regard to differentiation and our conversations with the FDA, as we have actually highlighted our potential differentiation, the mechanistic differences behind that, et cetera. But I think it's too soon to comment on where the FDA and where our discussions with the FDA on that.

Dae Gon Ha

Great. Thank you very much.

Operator

Debjit Chattopadhyay, Guggenheim.

Morning. This is [Rai Forcit] from Debjit team. Did the alignment with the FDA include any feedback on off-target editing profiling akin to sort of the AdComs criticism around cash generation characterization for the breadth of genetic diversity.
And our second question for the yet to be disclosed on program where you're going to offer primate proof of concept. What characteristics of this program are you most excited about the market size, the opportunity for first-in-class, the specific editing chemistry, et cetera?

Gilmore O'Neill

Thanks very much, Debjit. So um, let me actually pass the first question to Baisong.

Baisong Mei

Yes, that's where we have, but we have continuous engagement with FDA. So we are looking the engagement is scientifically driven this to understand the science, our molecule, that data we have and then and how the patient was manager.
And we are have a whole range of engagement with FDA and we know from preclinical CMC to clinical. So because, as I mentioned, we have Ahmed with destination and we have a lot of leverage and a lot of opportunity to actually engage with FDA. So we are very happy with the collaborative nature of the engagements.

Gilmore O'Neill

If I can I just add to that is that we, as we said before, when we actually watch the AdCom discussion. We were very gratified by what we heard and saw because our confidence at both in the comprehensive nature of our M. off-target editing oversight package was actually very robust at relative to the discussion of the AdCom.
And frankly, our off-target editing data package is actually very good and not surprisingly, because we are using our own engineered ASCA. 12 A. enzyme, which is a high fidelity as well as high-efficiency enzyme. And it's worth saying that in our in our hands and the hands of others on off-target editing is not detectable across the genome wide screen on as opposed to cast nine. So we feel very good about that.
And then with regard to the in vivo characteristics, I think I just want to say I said before that our key things are factors that we are focusing on is to select a set of targets that are high conviction based on the potential for critical differentiation from the current standard of care. And it actually are it does include a number of variables include the probability of technical success as well as regulatory success and arm commercial success.

Thanks for the rundown. Thank you.

Operator

Philip Nadeau, TD Cowen.

Philip Nadeau

Good morning. Thanks for taking our question or questions on manufacturing. Can you remind us where you are in the scale-up process, the commercial manufacturing scale-up process? And in your discussions with the FDA, have you agreed upon a CMC package? And in particular, is there a requirement for patients in Ruby to be dosed with the commercial material?

Gilmore O'Neill

Thanks very much for your question on from a CMC point of view, we actually are in a very good place. We have, as you know, it had discussions with the FDA and are actually progressing very well along that line we actually are and as you know, we have our building our commercial capacity and obviously that capacity will be ready for the demand that will exist at the time of our launch and is ready for supporting demand beyond that launch. And then with regard to the processes, I'd say we're making excellent progress there in support of our BLA so that we would be BLA ready and inspection ready it that.

Philip Nadeau

Thank you.

Operator

Jay Olson, Oppenheimer & Co.

Jay Olson

Hey, congrats on all the progress and thank you for providing this update. Our question is about your in vivo program. Can you talk about how and when you're planning to share preclinical proof of concept and since it seems like there's two undisclosed targets in your corporate deck. Are you planning to share preclinical proof of concept for both programs? And also any thoughts you could share on your choice for editing tool and delivery tool.

Gilmore O'Neill

And thanks very much. So from the in vivo pipeline point of view, with regard to the Highlander, when we're excited to be on track towards a POC this year for a 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, other form in which we would share at 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 Invivo this year.
Oh, sorry. With regards to the editing tools, well, we have, I think, been very clear that we are focusing on our ASCA. 12 A. 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 on and because we have human proof of concept, we have very exciting, robust editing data in human cells from a US customer today from our red cell program so all of those are the reasons why we're favoring and using that editing tool and that regards to delivery, we are using a nonviral focusing on nonviral delivery and nanoparticles specifically super helpful.

Jay Olson

Thank you so much.

Baisong Mei

Thank you.

Operator

Yanan Zhu, Wells Fargo.

Yanan Zhu

Oh, great. Thanks for taking our questions. Were just wondering about the midyear readout from the REVEAL trial. I think you have 18 patients dosed to date. Are you going to report data on? Are these 18 patients plus any additional patient dosed with a certain amount of follow-up at the time of readout.
And also seems like this is a much bigger number compared with the last readout. So I was just wondering how your confidence level of continued continuing to show the total hemoglobin normalization on kind of the goal in this media readout. Thank you.

Gilmore O'Neill

Thank you, Anna. And before passing to our base on some of the details, you know, 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 well above the 30% threshold that natural history would tell us is relevant, but we actually also see that consistent correction of anemia to normal physiologic range in all men or women treated and followed our past four months to date. I yes, we're excited about the midyear disclosure. But with that, I'm going to pass it to base on to give you a little more detail?

Baisong Mei

Yes. And thank you, Matt. 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 and plus more that we're going to be dosing before the end of the year. And as you can see, we yes, as you can see, we now publish data in ASH and some patients already over a year.
Then this station will have continued to monitor for the patient longer part of follow-up period and then we have 18 patient patient both now and then by the middle of the year. And this will help this patient is a patient. We have three or five months more of data by the time we release these that they will with. That's why we just described as really very meaningful substantive data will be shared midyear as exactly mentioned. We've got this data set is pretty strong.

Gilmore O'Neill

Yes. And we actually I think, based on I think you said it, but it is worth reemphasizing. We're talking about ranges of follow ups from an efficacy point of view between 3 and 18 plus months. So when you aggregate the total quarter patients now we are now really building not just a dataset that is robust in number, but actually as robust in follow-up period of time, which obviously relevant not just to our hematological and efficacy outcomes, but actually also increasing our confidence in durability.

Yanan Zhu

Great. Thanks I look forward to that.

Operator

Luca Issi, RBC Capital.

Luca Issi

Well, great. Thanks so much for taking my question and congrats on all the progress, maybe based on any update on the grade to polycythemia case that was potentially related to resell? I remembered a poster at ASH actually noted that causality of the disease was being investigated a 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 XU. S? Thanks so much.

Gilmore O'Neill

So based on we'll take that first question. And with regard to partnering, I will just say that we are keen on 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 you know, our focus is on at driving our at Cyclacel and Tata senior programs here in North America. And as I say, partnering will be something that we would look to in the future as upside with regard to the richer psychosis and basal.

Baisong Mei

Yes, thanks update that specific patient had a transient elevation of tourism globally. As we reported at ASH at the time of ASH, reporting is already Bapi normal for more than six months and then continue the patient about hematology parameters, including total globin, continues to stay normal. And then the investigator had a for the investigation of the patient data. And we do that too, and the patients the investigator consider this event is not related to the cycle or the vendors are treated.

Luca Issi

Yes. Thanks.

Operator

Steve Seedhouse, Raymond James.

Timur Ivannikov

Hi, good morning. This is Timur Ivannikov on for Steve Seedhouse. So we just had a clarification question on your PRO tools in Ruby. To what extent are you going to be using the same tools that Vertex and crisper use before like EQ VAS factory and BMT. four? And to what extent do you think of including new tools in on the issue being you're not going to be able to do a clean cross-trial comparison potentially here. Thank you.
Yes.

Baisong Mei

Thank you for the question. As I mentioned commented earlier, we used the tool from two and when is it more general, the quality-of-life tool as well as sickle cell specific. And the as you mentioned, specifically, the tool we're using.
We have a domain for our check that evaluate the fatigue application, which is important, not complain from a sickle cell patient, just give you example wise and you are very much of it is exactly the direction we were thinking trying to see.
Okay. What are the specific instrument? Be able to detect that a major complaint from the sickle cell patients such as fatigue, such as pan out and among other things.

Gilmore O'Neill

So we're actually using a number of instruments. Some were used by Vertex, and we have additional instruments in our PRO armamentarium, and they are actually being collected in the Ruby Phase one two three trial as we speak.

Operator

Jack Allen, Baird.

Jack Allen

Great. Thanks so much for taking the question and congratulations on the progress. I wanted to touch a little bit on the patient experience with Fermacell. Have you provided any disclosures around the number of inquiries cycles that are required to receive from the sale.
And the and I was wondering if the higher effected editing efficiency of Castleberry allows for a shorter amount of recent cycles? And then on the back end after treatment, what are you seeing as it relates to time to neutrophil engraftment on that? And how do you think that comparison some of the competing products in the space.

Gilmore O'Neill

Thanks so much and thanks very much, Jack. I'm actually going to ask the song at talk about the clinical experience with varices and other elements of the patient experience and obviously touch on news from the graph and with which we have been very pleased to date.

Baisong Mei

Thank you, Jack. We out for patient experience, I mean versus a certainly a very important part of that. As I mentioned earlier on since I joined, I work with the team and experts, we amended the protocol and trying to optimize the free seats process.
So now we are very happy with the number of cycles that a patient has been gone through, and we can see that's already improvement from what we had before in terms of the the engraftment, as we disclosed that in ASH and we have all the patients have engraftment is under within 30 days. So we're very happy about that. And we continue to see the similar data as we follow through the protocol for our follow-on studies, if that's okay.

Jack Allen

Thank you very much.

Operator

We have reached the end of our question and answer session. This concludes today's conference. Thank you for your participation. You may disconnect your lines at this.