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Intrinsic Value is all-important and is the only logical way to evaluate the relative attractiveness of investments and businesses.
Intrinsic Value is all-important and is the only logical way to evaluate the relative attractiveness of investments and businesses.
Good morning, and welcome to Editas Medicine’s First Quarter 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.
Thank you, Camilla. Good morning, everyone, and welcome to our first quarter 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 its 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 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.
Thank you, Cristi, and good morning, everyone. Thank you for joining us today on Editas’ first quarter earnings call. I’m joined today by two other members of the Editas executive team, Baisong Mei, our Chief Medical Officer; and Michelle Robertson, our Chief Financial Officer.
As many of you know, in early January, we shared our strategy to position Editas as a leader in, in vivo programmable gene editing and hemoglobinopathies. During the first quarter, we successfully executed this strategy driving to our goal of delivering life-changing medicines to patients with previously untreatable or undertreated diseases. We are increasing our momentum in driving our ex vivo EDIT-301 program as we pursue a leadership position in hematopoietic stem cell medicines for hemoglobinopathy.
As a quick recap, there are three underlying pillars to our new strategy.
First, while continuing to develop EDIT-301 for severe sickle cell disease and transfusion-dependent beta thalassemia, or TDT, we have sharpened our discovery focus to in vivo administered genome editing medicines. As part of that refocusing effort, we previously announced that we had divested our iNK cell franchise to Shoreline Biosciences in January.
Second, we are strengthening our discovery engine and technological capabilities. We have divided our research division into separate technology and drug discovery groups, enhancing the capabilities of each and implementing our new target selection criteria.
Finally, our third strategic pillar is an increase in expanded approach to business development. In tandem, we will continue to deleverage our IP portfolio to drive out-licensing and partnership discussions.
So, how have we executed against our new strategy in the first quarter? We have increased our investment in our EDIT-301 program after reviewing promising initial RUBY Phase 1/2 study data that indicated that we have a competitive and potentially differentiated program to treat sickle cell anemia and TDT.
Additionally, we are investing to develop an in vivo approach for editing hematopoietic stem cells for the treatment of sickle cell disease and TDT, leveraging the unique and differentiated approach of EDIT-301 that we have already seen POC for in humans.
We continue to ramp up enrollment and dosing of patients in the RUBY trial for sickle cell disease and are on track to have dosed 20 total patients by the end of 2023. We are also excited to share that the FDA recently granted orphan drug designation to EDIT-301 for the treatment of sickle cell disease, and we are pleased to announce that in June we will provide a RUBY clinical data update in an oral presentation at the European Hematology Association, or EHA Congress, and in our company-sponsored webinar. Baisong will share further details regarding our June data readout and our enrollment progress in his remarks.
On EDIT-301 for TDT, we are pleased to share that we dosed the first patient in our EDITHAL Phase 1/2 trial in the first quarter and that the patient has successfully engrafted neutrophils and platelets. Enrollment continues to progress and we remain on track to provide initial clinical data from the EDITHAL trial by year-end.
Moving to in vivo. Earlier this year, our drug discovery group began lead discovery work on in vivo therapeutic targets at HSCs or hematopoietic stem cells and other tissues. As a reminder, under our new target selection criteria, we will select therapeutic targets that will allow our genome editing approach to differentiate maximally from the current standard of care for serious diseases. The target selection criteria will work to ensure targets are selected that maximize the probability of technical, regulatory and commercial success. Our search for a new CSO to lead this drug discovery group continues to progress, and I look forward to updating you on this search and our in vivo work in the future.
Turning to our intellectual property position. Since the founding of Editas, we have placed substantial importance in securing robust intellectual property protection, covering our cutting-edge scientific discoveries and gene editing advancements to enable the development of novel transformative medicines for patients in need. It is important to note that we have a large portfolio of foundational U.S. and international patents covering CRISPR/Cas9 in human therapeutics, only some of which are subject to interference proceedings. And we are confident that our IP portfolio will provide meaningful value in the future. We are the exclusive licensee of Harvard University’s and Broad Institute’s Cas9 patent estates, and Editas is uniquely positioned to issue exclusive and nonexclusive licenses for Cas9 to any company seeking to use these enzymes to make human medicines, including an in vivo and ex vivo therapeutic applications.
Our unique position as the exclusive licensee of this patent estate ensures that we are the party responsible for any licensing discussions as CRISPR/Cas9 products enter the market, which, given the size of the U.S. patient market, the number of companies buying to develop CRISPR/Cas9 medicines is a substantial position.
With our annuity sharpened strategic focus, our world-class scientists and employees and our keen attention to execution, we continue to build upon the momentum from our clinical readout milestones during the fourth quarter of 2022. We look forward to updating you on our progress and on the execution of our new strategy throughout the year.
Now, I will turn the call over to Baisong, our Chief Medical Officer. Baisong?
Thank you, Gilmore. Good morning, everyone.
Let’s start with EDIT-301 RUBY study for severe sickle cell disease. As Gilmore mentioned, we continue to enroll and dose patients in the RUBY study. We have activated 20 study sites and enrolled 19 patients, almost double the number of patients enrolled from three months ago. As we previously shared, we began parallel dosing of patients earlier this year. We are on track to provide an update on the RUBY clinical data both next month and year-end as well as to dose 22 total patients by year-end.
Turning to clinical data. I’m excited that we will present RUBY clinical data as an oral presentation at the European Hematology Association, or EHA Congress, and at our company-sponsored webinar in June. The data set will include safety and efficacy data for multiple patients, including 10 months data from the first patient treated and 6 months data from the second patient treated, including total hemoglobin, fetal hemoglobin. We will also share data on safety, neutrophil and platelet engraftment and vaso-occlusive events or VOE, from the first four patients.
As a reminder, last December, we presented initial data from the first two patients treated in the RUBY trial. The first patient who had five months of follow-up after treatment with EDIT-301 showed clinically significant improvements across all hematological parameters and no VOEs. Specifically, that patient had an increase of fetal hemoglobin fraction to 45.4%, five months after EDIT-301 infusion. And the correction of anemia with total hemoglobin level well into the normal range at 16.4 grams per deciliter.
These initial clinical data indicated that EDIT-301 provides patients with high and sustained level of fetal hemoglobin and normal level of total hemoglobin. This clinical observation is consistent with preclinical data, which has demonstrated that targeting of gamma globin promoter enables increases of fetal hemoglobin independent of erythropoietic stress.
Given the unique gene-editing approach and mechanism of action by EDIT-301, supported by preclinical data and initial clinical data, we continue to believe that EDIT-301 can potentially provide robust clinical benefit to patients with severe sickle cell disease, and potentially provide clinical differentiation in the long-term.
As a reminder, a sustained normal total hemoglobin level is an important clinical outcome for patients, as the correction of anemia can significantly improve quality of life and ameliorate an organ damage. We believe sustained normal level of total hemoglobin could be a potential point of differentiation for EDIT-301.
Turning to EDIT-301 EDITHAL Phase 1/2 trial for transfusion-dependent beta thalassemia. As Gilmore mentioned earlier, we dosed our first patient in Q1 and the patient has successful neutrophil and platelet engraftment. We remain on track to provide initial clinical data from the EDITHAL trial by year-end.
As we have done for the RUBY study, we are also taking multiple measures to accelerate the development of EDIT-301 for TDT and have strong positive momentum. We have enrolled multiple patients who have completed pheresis and have their CD34 positive cells edited or are in the process of pheresis.
Recently, I have been traveling around the country visiting our RUBY and EDITHAL clinical trial sites. I very much appreciated the enthusiasm and the support from the investigators and study sites. I’m pleased with the momentum of EDIT-301 in patient recruitment, pheresis, editing and dosing in both studies.
I’m excited to hear from investigators that patients dosed with EDIT-301 have already seen positive changes in their lives. We look forward to sharing additional updates as the year progresses, including RUBY study data next month and at year-end, and sharing initial clinical data from EDITHAL study by year-end.
Now, I will turn the call over to Michelle, our Chief Financial Officer, to review our financials.
Thank you, Baisong, and good morning, everyone. I’d like to refer you to our press release issued earlier today for a summary of our financial results for the first quarter of 2023. I’ll take this opportunity to briefly review a few items.
Our cash, cash equivalents and marketable securities as of March 31st were $402 million compared to $437 million as of December 31, 2022. We expect our existing cash, cash equivalents and marketable securities to fund our operating expenses and capital expenditures into 2025.
Revenue for the first quarter of 2023 was $9.9 million compared to $6.8 million in the same period last year. The increase is related to the previously announced sale of our oncology assets to Shoreline Biosciences and related licenses, which was completed in January 2023.
G&A expenses for the quarter were $23 million compared with $19.5 million for the first quarter of 2022. The $3.5 million increase is primarily attributable to increased professional services expenses to support business development activity, partially offset by a decrease in stock compensation expense.
R&D expenses this quarter were $38 million, which was flat compared to the first quarter of 2022. This reflects a decrease in expenses following the strategic reprioritization of our portfolio, offset by increased investments to accelerate the development of EDIT-301. This reallocation of capital is in line with our strategic priorities.
Overall, Editas remains in a strong financial position, and our sharpened discovery focus allowed us to concentrate our talent and extend our cash runway into 2025, which provides ample resources for our continued progress in both of our EDIT-301 trials as well as advancing our research efforts in hemoglobinopathy and other in vivo discoveries.
With that, I will hand the call back to Gilmore.
Thank you, Michelle.
It has been almost one year since I joined Editas. In this time, the Company has demonstrated two clinical proof of concepts, including a proof of concept for EDIT-301, which has the potential to be a competitive and differentiated product for the treatment of sickle cell disease and transfusion-dependent beta thalassemia. In addition, as I stated in my opening remarks, we’ve taken a number of tangible steps to reshape the Company around our new strategy, which we shared in early January and have begun executing on that strategy. And this is just the beginning. We look forward to continuing our transformation on sharing our progress with you.
As a reminder, our strategic objectives for the year include providing clinical updates from the EDIT-301 RUBY study in June and end of 2023, providing clinical data from EDIT-301 EDITHAL trial for TDT by the end of 2023, dosing 20 total patients in our EDIT-301 RUBY study by year-end, hiring a new CSO with specific expertise aligned to our vision, advancing discovery of in vivo editing of hematopoietic stem cells and other tissues, and finally, leveraging our robust IP portfolio and business development activities to drive value and complement our gene editing technology capabilities.
I thank all the patients, investigators and our employees who are helping to drive our strategy forward. Thank you very much for your interest in Editas, and we’re happy to answer questions. Thank you.
[Operator Instructions] And our first question comes from Joon Lee with Truist Securities.
Hi. Thanks for taking our questions. Novartis recently terminated their sickle cell disease program after treating a couple of patients to see no benefit. Given your editing strategy is similar to what Novartis said, could you point to some differences why your promoter editing strategy should continue to work when Novartis failed? And I have a follow-up. Thank you.
Thanks very much, Joon. Yes, we did actually see that event some weeks or months ago. I think there are some elements that are critical. I think the first thing to point out that -- is that in our initial POC readout at the end of last year, we actually saw a very robust data with increase in total hemoglobin -- early increase in total hemoglobin as well as a robust fetal hemoglobin expression completely consistent with the preclinical data that were generated.
In testing our preclinical and our clinical hypotheses that our unique approach of combining an AsCas12a effector CRISPR enzyme with the targeting of a different region of the HBG1/2 promoter, which was much closer, in fact, actually encompasses the area in which we see deletions or mutations associated with hereditary persistence fetal hemoglobin. I think we believe that all those factors point towards a key difference and differentiation, and indeed, our preclinical data and our clinical data have actually supported that hypothesis.
So, can you remind me if you have any data preclinical? Having -- comparing the editing -- the same region in Cas9 versus AsCas12 that you’re using, and what the difference is maybe?
Sorry, Joon...
Can you…
Yes. So I mean, comparing Cas9, which is what I think Novartis used and AsCas12a, which is what you’re using, you can have different outcomes if you were to target the same region in terms of getting deletions or…?
Joon, I think you are a little breaking down, but let me try. This is Baisong. Let me try and see whether I understand correctly. There is background noise. So I think your question is to say compared to Novartis and do you have a comparison between Cas9 and Cas12a and also the region just Gilmore mentioned. Is that your question?
Yes. No, actually, if you target the same region, either with Cas9 or Cas12a, what differences in outcome you get?
Yes. Let me just kind of take it. We did the comparison, we did -- from a clinical perspective, we scanned a large region -- of the promoter region to identify which areas to do the editing. So, without too many specifics, but we covered a large region of the promoter in the HBG1 and 2. And we find out that related to the region we selected, which Gilmore just mentioned, is consistent to the clinical observation for these HPFH. And then we also compared the Cas9 with Cas12 and we found the difference between Cas9 and Cas12. Does that answer your question?
Yes. No, absolutely. Your impaired data is very good, but just was just curious what was driving that difference. Thank you so much. I’ll hop back in the queue.
Thank you.
Our next question is from Samantha Semenkow with Citi.
Just a couple for me. For the presentation at EHA, is that a late-breaking presentation? I just wanted to clarify.
Thanks, Samantha. I’ll just have Baisong update you or give you detail there.
It is a normal oral presentation that is expected.
And then I also wanted to clarify, I heard you mention, obviously, we’ll have updated data for the first and second patients. And then, I heard you, I think, say, four patients. So will it be six patients total that will get data on VOE, or is that -- or VOCs, or is it four patients total?
Total will be four patients at the EHA presentation.
And then when you’re making that cutoff for those incremental additional two patients, what level of follow-up was the cut-off there? So I’m just curious, is it a couple of months, is it one month? Any information you can provide would be helpful.
Yes. We are -- for these next two patients, we’ll have two months or more.
Just a follow-up, one other thing, I think it’s important to understand that, obviously, we will, because the abstract, which will be published later, was based on data cut earlier, we will actually be presenting more data than in the abstract at the EHA Congress.
Yes. Thanks, Gilmore. And just kind of also follow-up on that, the abstract will be available on May 11th.
Next question is from Dae Gon Ha with Stifel.
Hey, great. Thanks so much for taking the question. And look forward to the data update next month. So, I guess, I was just kind of wondering about your strategy going forward. So, maybe if you can kind of walk us through how you’re thinking about next programs or priorities beyond EDIT-301? I think, Gilmore, you mentioned in vivo HSC editing. But curious, is delivery tech or less burdensome conditioning a stronger emphasis in your lineup, or is it advancing new programs? And if it’s the latter, I guess, would you continue to do other ex vivo HSC, or is it more of an in vivo? And in that case, would you also think about other organs? Then I’ve got a follow-up.
Yes. Thanks very much, Dae Gon. We are -- the large part of our discovery focus is actually on in vivo. I think that was a very important part and pivot of our strategy because we believe that it maximizes the -- or maximizes our ability to exploit the powerful technology that we have available to us.
From a point of HSCs, if you reduce the problem of in vivo to sort of three elements, selecting a robust effector molecule or enzyme, CRISPR enzyme, selecting a good target and then delivery, we believe that we have solved two of those problems, with very robust human data in the use of our Cas12a, CRISPR enzyme and the target of that specific HBG1/2 promoter. And so that reduces it to an in vivo delivery problem.
As I said in my earlier remarks, our discovery group is actually working on that, and we look forward to updating more at an appropriate time in the future. I will say that we are looking actually also beyond HSCs to other tissues. And again, we’ll give further updates in the future.
And then second question, I just wanted to follow up on Baisong’s commentary during prepared remarks. So, as you were going into the field and kind of gauging physicians’ take on EDIT-301, you expressed -- or you commented on their high enthusiasm. Wondering if you could comment, I guess, what proportion of those docs you visited are also looking to administer CTX001? And I guess, has there been any kind of gauge or ascertainment from your part as to what their sort of motivation would be in taking CTX001? Like, are they lining up patients right now for CTX001? What kind of sentiment do you have? Are there any reservations on that approach? Any thoughts on that would be helpful. Thanks so much.
Yes. Thanks for the question. Yes, I visited quite a few number of study sites. Actually, many of them are being -- participating in the previous gene editing trials. So, they are very enthusiastic about the approach we are taking, including the different targeting region for editing and the different enzymes to do. And so, actually, the benefit for us is those investigators have a lot of experience in this field.
Yes. I think just building on Baisong’s remarks, as you’re obviously looking out towards the evolution of the market against the background of enthusiasm for our investigators and indeed the patients with the increase in our acceleration enrollment. We anticipate that in the future that the vast majority of patients will be awaiting dosing at the time of our launch, and I can go into more details on that.
But I think, a very important point, I think something that has really resonated with the investigators is that our initial clinical data were very encouraging as presented in December, consistent with our preclinical data, and we’re actually very confident that we will see replication in subsequent patients as we continue to monitor them through the execution of the 301 studies.
Our next question comes from Steve Seedhouse with Raymond James.
My question actually requires a bit of a prelude, so I hope everyone can bear it for a moment. You made a comment on globin locus editing increasing fetal hemoglobin independent of erythropoietic stress. And as you know, ICER -- the recent ICER report on exa-cel uncovered an ongoing phlebotomy, at least one in sickle cell and some of the earlier data releases for that program in thalassemia indicated phlebotomy use there as well, but that just stopped being reported at some moment. So it’s not clear how prevalent phlebotomy use is for exa-cel. And this is all important because there was data at ASH years ago, as I’m sure you also know, indicating BCL11A editing cooperates with phlebotomy and primates to accentuate F cells and ultimately HBF levels probably because of the stress erythropoiesis causes.
So all that said, I’m curious if you agree that phlebotomy is potentially confounding fetal hemoglobin data for BCL11A approaches. And if you know what is the impact specifically where your editing approach at the HBG1/2 locus, what has phlebotomy use been like in your study and if you think this is all potentially a competitive advantage for you? Thank you.
Steve, thank you very much for your question. So, from our own clinical data, preclinical data and also published clinical data, you’re probably referring to for the BCL11A targeting approach. It did require some stimulation for the erythropoietic stress to increase the fetal hemoglobin, sufficient fetal hemoglobin expression. And so, that’s actually the reason we’re treating the target we are treating now, and we actually did take longer time to get all the targets from a preclinical study perspective, and that’s been validated by other publications.
And regarding specific clinical data for the BCL11A approach, I have not seen formal publication. So, I would be waiting for them to publish their data and so we’d have a better understanding. So I will not comment on their data unless published.
However, it is worth pointing out that in our early disclosure, we actually noticed the combination of a very robust normalization or correction of anemia in our first patient in December. And that was associated with a robust fetal hemoglobin expression, suggesting that indeed stress erythropoiesis as we hypothesized based on the known biology and our nonclinical data, that our approach is not dependent on erythropoiesis.
Our next question is from Yanan Zhu with Wells Fargo.
Maybe to continue the discussion a little bit from the prior question. Gilmore, you mentioned that the total hemoglobin for the first patient is quite robust in the normal range. The percent of fetal hemoglobin appears to be very much in line with competitor gene editing product. So, I was wondering, is the -- greater total hemoglobin reported for that patient, is that due to the total number of red blood cells? Could that potentially be a reason, or could it be related to the baseline level of hemoglobin in that patient?
And along that line, to continue a little further and to -- perhaps looking into what we could expect from patient number two at EHA. I was just wondering, could you remind us the baseline hemoglobin for patient number two and what is the normal range for the female patient, which obviously is the second patient? Thank you.
So thanks for the question, Yanan. So, I will start with answer to your first part of the question regarding the fetal hemoglobin versus the number of red blood cells and all these. So, what we see is actually robust erythropoiesis for these patients we observed. So, their hemoglobin level is contributed by both the hemoglobin per cell as well as the number of red blood cells. And you can see that -- we actually do see the increase also on both ends of that. And then also I want to mention that even though we also have like around 45% of fetal hemoglobin, and because of the total hemoglobin level is high and the total amount of fetal hemoglobin is also high. So, that’s kind of in that.
And then regarding the second patient, and we will present the data very soon. So, I will not comment on the specifics of the patient data, but I can mention that, of course, the male and female normal hemoglobin level are different. Usually, for male is around 13.5% to up to 18-gram per deciliter, for female is around 12 to 14, depending on the reference lab. So there’s some difference in there, too.
Okay. I think one other thing, Yanan, you asked a question about what was the baseline hemoglobin of the patient one. And I think what we can say is that the hemoglobin or the total hemoglobin increase that we saw occur very rapidly just within the first few months of dosing comprised a 3.5 gram per deciliter increase.
Yes. Maybe just add a little bit nuance on that baseline for sickle cell patient, especially for gene-editing trial. And because the patient -- when they prepare for pheresis and conditioning, they usually have blood transfusion. So, the baseline actually we have is not the lowest level we recorded. We just set at the time of the visit 2 as a baseline. So actually it’s compounded, it could be many different reasons for the baseline on that, too. That’s just a nuance. It’s actually -- it’s lower than -- it’s around a little bit over 10 grams per deciliter when we actually -- on our record for this patient. This is just an example about the baseline, maybe not confusing.
Very nice. Thank you for all the explanation. Maybe a quick follow-up. Do you expect this to be also a differentiator for TDT and perhaps maybe at a greater level of significance because anemia is a major manifestation of that disease? Thank you.
We designed our discovery group and scientists designed and selected the combination of Cas12a with the specific targeting of the HBG1/2 promoter using a set of empirical experiments to determine what was the best approach to driving not just fetal hemoglobin expression, but robust erythroid output that would be -- or red cell output for the bone marrow that would be independent of erythropoiesis or anemia. And those empirical experiments really determined that approaching or directly targeting the HBG1/2 promoter would be better. And that was the original design hypothesis.
The nonclinical data -- preclinical data actually supported that, showing robust erythroid output in comparison to other approaches as well as robust fetal hemoglobin expression. And indeed, that is what we have seen as we disclosed in our first sharing of the data or cut of the data from our RUBY study. And so, obviously, it is -- we haven’t seen enough data in our EDITHAL patient. But what I can say is that RUBY has certainly demonstrated data that are consistent with both, the preclinical data, which were supportive of the original biological and therapeutic hypotheses.
Our next question is from Phil Nadeau with TD Cowen.
This is Ernie Rodriguez for Phil. Thanks for taking my question. On the on the sickle cell program, have you met with the FDA to gain better visibility on the regulatory path? And then, a second question on the TDT program. For the year-end update, would that include only the sentinel patients that you initially dosed, or will you disclose additional patients? And if you will be disclosing additional patients, are you planning on reporting when you switch from sentinel dosing to parallel dosing?
So I think what I’ll do is ask Baisong. I’ve actually -- I’ll keep track of the question. Baisong, if I’d ask you just to answer the question on the FDA and the regulatory interactions.
Yes. Thanks for your question. So, we certainly have a lot of engagement with FDA. As you see that recently, we have the orphan drug destination. And from the registration perspective, we previously announced that we actually have the alignment on the potency assay with the FDA, which FDA will consider this efficacy data can be supporting registration. And we will have further engagement with the agency to align on the total registration package for the BLA submission, which is also planned.
And your second part of the question is about the beta-thal data. So, we are moving really along with the EDITHAL study, and we expect that we will have data by year-end, more than sentinel patient. And so, we’re looking forward to share that data by the year-end.
And you are -- more than sentinel patient, so are you planning to disclose when you going approve to continue parallel dosing before then?
So, what we are actually planning to do is -- I think the key thing is we’re on track to get the data for readout at the end of the year for that initial readout at the end of the year. We haven’t determined if we’re actually going to share that. But I think the important point is that we are well on track to disclose good initial data for the end of the year.
Our next question comes from Rick Bienkowski with Cantor Fitzgerald.
I guess, I’ll expand a bit on the last question on the path towards registration for EDIT-301. 20 patients is a pretty substantial cohort size in sickle cell disease. So, do you have any sense of how many patients’ worth of data you will need for a registrational filing?
Yes. Thank you for the question. So we certainly think that with the gene editing approach that we have we will be able to generate a substantial amount of data. And the specifics on the number of patients to be able to use for registration, we need to align with the regulatory agencies. So, we are planning to discuss with FDA.
And I just have another quick one. I was hoping for a little bit more granularity on the collaborative revenues for the quarter. Were all of the $9.9 million in revenue attributable to the Shoreline transaction, or are there some other revenues attributable to other partnerships in there?
It’s a combination of both the Shoreline and then some other small sublicense revenues.
Our next question is from Rich Law with Credit Suisse.
I have a couple of questions for you guys. So, with the appeal litigation pending, what does it mean for company such as CRISPR Vertex that already filed a BLA for exa-cel that utilize CRISPR/Cas9 from your IP perspective. They don’t have a license from you or the Broad and could potentially launch the product before we know the outcome of the appeal. So any insight here would be helpful. And then, I have a follow-up question.
I think the key thing is that we sort of anticipate the judgment in the early to mid-2024. We are confident that we prevail as we have before, largely because what are -- under discussion is the application of the law and not about new facts, and it’s the application of law by PTAB. Now setting aside that interference, I think the important thing is to say that we have a portfolio of IP not subject to any interference that actually covers product in development using Cas9 for the application of human therapeutics. And, looking forward, we are happy to grant licenses to enable delivery of this technology to patients, and believe that we should recognize significant value around that.
So, in terms of granting license, so we’re not going to know the appeal decision likely before the BLA and also potentially the launch? Like, how do you sort of think about that?
Well, I think there are a number of important points to make. I think the first, again, is just to remind that the appeal applies to some of our Cas9 in-human therapeutics IP estate, but not all. I think it’s important to emphasize that we have Cas9 or IP around Cas9 use in human therapeutics that is not subject to any interference, and therefore, is not subject to that appeals case. And we actually believe that it actually covers product in development. And so, what I think I want people to really understand is that that appeals case is around interference on some of our IP estate, but not all.
And then, just one more question for me. So you’re seeing some next-gen FCB [ph] therapies already in development with new conditioning agents. So, if those exceed, it doesn’t seem like the shelf life for these first gen therapies will last too long. Any thoughts about this?
So just to be clear I understand your question. You’re actually questioning if the evolution of new conditioning would actually change the landscape for the products that are either -- are close to approval? I think it really very much depends on the nature of the conditioning. As we look out at toxification, obviously, it’s something we’ve looked at closely. One of the important things is to balance both the reduction of toxicities with engraftment efficacy. And I think we all see that it’s a very important path to increasing eligibility for patients because more patients will be able to tolerate a non-genotoxic, less toxic conditioning regime. Many of the regimes or some of the approaches are not actually editing dependent. They are actually -- and so what we actually believe is that the evolution of milder conditioning could actually expand and grow the size of the eligible patient population for all.
I think, the important thing, obviously, also is that we are looking beyond, not just conditioning, but looking to in vivo editing as part of our strategy, for the very simple reason that we believe that in vivo editing will further increase the eligibility of the patient population for treatment.
Our next question is from Debjit Chattopadhyay with Guggenheim.
This is Ray Forseth on for Debjit. I just want to build off of the conditioning discussion and sort of get an outline of your strategy. Is it sort of bifurcated kind of exploring both in vivo editing and the opportunity to in-license assets that would be alternative to busulfan. Can you sort of map that out for us? And just wanted to get your thoughts, too, on the ASGCT abstracts and sort of what you see in the competitive landscape around conditioning, especially given that competitor is kind of moving forward with the CD117 approach?
Yes. Thanks very much, Debjit. I think I’ll start and then I will have Baisong comment. From a strategic point of view, we are using a two-pronged strategy. We have directed investment, significant investment internally to our discovery of in vivo editing for hematopoietic stem cells. And I think, as I said earlier, we believe that this is a problem that we can focus on, where we can focus on delivery, where certainly in humans, we believe we have solved the, two of the three challenges around the selection of a CRISPR enzyme as well as a target.
In parallel, we actually are continuing -- have ongoing evaluations of milder conditioning approaches. And I think you asked more importantly, or in follow-up a question about the, for example, CD117. I’ll ask Baisong just to talk about that.
Thanks for your question. I can say that we have looked into this milder conditioning in very deep, looking the space as well as internal efforts wise. And there were generally probably two approaches. One is that CD117 antibody and in that direction. And the other one is actually doing the cell modification together with gene editing. So, the latter approach is still -- is in infancy, if I may say, and the previous approach with antibody have many different exercise on that. So I think we are very closely monitoring the space and understand these. And I also want to mention that the milder conditioning, if successful, is not going to be only successful for sickle cell transplant, it’s going to be successful for leukemia and many different gene therapeutic areas. So, we are actually very much looking into this space.
Our next question is from Madhu Kumar with Goldman Sachs.
This is Rob on for Madhu. We were just wondering how should we think about forward OpEx, given -- forward OpEx, particularly R&D, given the robust recruitment into RUBY? And how long will spend be around cell editing and transplants versus follow-up?
I’m sorry, Rob, I actually had great difficulty hearing your question. Could you just repeat it, please?
Sure. We are just wondering how we should be thinking about forward OpEx, particularly spending in regard to transfusions versus follow-up?
Okay. So, I think you’re asking -- I want to make sure, you’re asking about forward-looking OpEx around the execution of the RUBY study. Is that correct? Okay. Michelle?
Yes. So, Rob, I mean, we don’t disclose our annual OpEx -- or our quarterly OpEx, but I can tell you that about half of our spend is in -- on the both the RUBY trial and the TDT trial. So, we don’t expect an enormous increase quarter-over-quarter. But as we do dose more patients, obviously, our R&D spend will go up, but not substantially. And our current, I’ll just say one more, our current cash runway supports our RUBY trials.
Our next question is from Greg Harrison with Bank of America.
This is Mary Keith on for Greg. Thanks for taking our questions. So, with 19 patients enrolled and plans for 20 to be dosed by year-end. Maybe how many sickle cell patients have been currently treated with EDIT-301, and maybe how could we expect to see this represented in the efficacy readout by the year-end update? Thank you.
So, we have 19 patients enrolled. And among those, as we just mentioned, 4 have been dosed, and we actually have -- more patients have been completed pheresis, and have CD34 cells edited and ready to schedule dosing. And then, we have other patients who are in the process of pheresis. And so we are very confident that we will be able to dose 20 patients by year-end.
Our next question is from Luca Issi with RBC Capital Markets.
Maybe on beta thalassemia, obviously most patients are in Southern Europe, so wondering if you could comment on what’s the plan to capture that market. And maybe how you’re thinking about some of the key lessons learned from the unsuccessful launch of bluebird bio there? And then, maybe on sickle cell disease, wondering if you can comment on pricing. Obviously, report suggests $1.9 million. So, wondering if that is actually aligned with your thinking. And then, maybe lastly on LCA10, any update on partner discussions there?
Thanks very much, Luca. So obviously, beta thalassemia is a disease that dominates parts of the world, particularly Europe, Southeast Asia, South Asia, amongst others. From a point of view of our forward-looking, we are actually focusing our efforts on North America currently. We have shared in the past that from an upside point of view, we are looking -- open to partnering, ideally targeting as a partner with a large global footprint who would actually collaborate certainly in sort of ex-U.S. development and commercialization. So that’s what I would say I can talk about when we look to your point about beta thalassemia outside North America. I will say that we are happy -- very happy with the progress that we’re making with execution here within the United States and North America.
With regard to pricing, I think it’s very early days yet for us to be talking about pricing. This is something that we would be very happy to discuss when we’re actually closer to approval and launching, and we’ll look forward to future conversations around there. Obviously, we look to the market evolution over that time, but we’re going to talk about that closer to launch and approval.
And then, finally, with regard to LCA10, we have -- we really have a practice of not really going into details until we have a deal signed and executed.
And maybe just add one more point -- sorry, just add one more point. As Gilmore mentioned about pricing, right? So certainly, we are very early stage, and we are happy to see the community is looking to the value of this gene editing therapy, and we are happy to see that the report recently in this space. So we -- as Gilmore mentioned, this will be evolving, but we are pleased to see that -- the entire community recognize the value of the medicine in this field.
Our next question is from Jay Olson with Oppenheimer.
Good morning. This is Chang on the line for Jay. Maybe two from us. So, I’m just wondering if there’s a chance where you have the capacity to dose more than 20 patients for the sickle cell disease program this year. And second question is on your ex-U.S. strategy. What you’re kind of thinking? And if you are planning to partner that program, what is the best timing to do that? Thank you.
Thanks very much. So, do we have capacity to dose more than 20 patients? Yes. One of the important points, when we rolled out our strategy was to again sharpen our focus on developing and accelerating 301. And indeed, we have deployed capital to enable, not just the acceleration on the clinical side, but actually also to ensure that we have capacity to edit or other CMC capacity to edit and support those, that clinical acceleration. So indeed, we do have that capacity to dose more than 20 patients.
And then, I think your second question was around ex-U.S. and the timing of partnership. I think as I said before, we are interested in partnering. We’re looking to a partner with a global footprint that would actually certainly support ex-U.S., particularly on the development and commercialization. And with regard to timing, we wouldn’t really discuss the timing until we actually have a deal signed and executed.
Our next question is from Joel Beatty with Baird.
Hello. This is Benjamin on for Joel. Thanks for taking our questions. Looking across other late-stage products in sickle cell and TDT, it appears that data sets of 30 patients could support approval. So, with Editas being on track to dose 20 patients by year-end, how quickly do you think you’ll be able to secure the necessary data to support regulatory approvals? Thank you.
Yes. Thanks very much, Ben. Baisong?
As I mentioned earlier, in terms of total number of patients required to support registration and then need to have required alignment with the regulatory agency. And in terms of the progression of the study, we are very positive about the momentum. So, we are very optimistic we will be able to dose patients as we planned.
So, I think the key thing, Ben, is that you’ve actually identified sort of a benchmark. But obviously, what we need to do is, as planned, sit with the regulators and come to an agreement on what the data set they would like to see for our programs.
Our next question is from Joon Lee with Truist Securities.
So, I had the same question as Steve, but maybe a different way of asking. What percentage of sickle cell patients with hereditary persistence of fetal hemoglobin have mutations along the globin locus versus the BCL11A locus?
So, we do not have all the specifics on your question on that, but what we know is the mutation, the promoter region directly impacted the fetal globin expression. But BCLA is a transcription factor, which impacts multiple different cells in it. And the mutation of the BCL11A will have much different impact from the fetal globin expression only, will have other impacts, too. So the gene optimization [ph] is a much more complicated issue than the HPFH with the promoter region and mutation for the gamma globin promoters.
I think another way, Joon, to actually characterize this because some of the prevalence is a little harder to quantify, but another way of pointing it is really the strength of the signal, the hereditary persistence of fetal hemoglobin and its capacity to substantially mitigate the effect of sickle cell disease and thalassemia, were actually determined actually quite some time ago because this phenotypic change -- this phenotype to genotype correlation was actually quite robust and identified actually a few decades ago, whereas the BC11A was identified really through a GWAS assessment, genome-wide assessment.
Thank you. We have reached the end of the Q&A session. And with that, ladies and gentlemen, this concludes today’s call. Thank you once again for your participation. You may now disconnect your lines.