Q4 2022 Editas Medicine Inc Earnings Call
Speaker 1: Good morning. Welcome to Editas Medicine's fourth quarter and full year 2022 conference call. All participants are now in 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.
Speaker 1: I would now like to turn the call over to Ron Moldaver, Invest Relations for Editas Medicine.
Speaker 2: Thank you, Sherry. Good morning, everyone, and welcome to our fourth quarter and bolear 2022 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 on the Investors section of our website approximately two hours after its completion. After our prepared remarks, we will open the call for Q&A.
Speaker 2: 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.
Speaker 2: 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...
Speaker 2: by two other members of the Editas executive team, Beisong Mai, our Chief Medical Officer, and Michelle Robertson, our Chief Financial Officer.
Speaker 3: Last year was a landmark year in EditHass Medicine's history as our team demonstrated two clinical proof of concept in patients using both in vivo and ex vivo therapeutic approaches. In November , we announced data demonstrating human proof of concept for our in vivo Edit101.
Speaker 3: A.V. delivered Cas9 therapy for LC-10 and inherited retinal disease. However, due to the limited addressable population, we made the tough decision to pause patient enrollment in our bridge trial. In December , we shared very encouraging initial data from the Ruby Phase 1-2 clinical study of our ex vivo autologous ed. 301 therapy in severe sickle cell disease.
Speaker 3: as we continue to drive towards our goal of delivering life-saving medicines to patients with previously untreatable or under treated diseases. As many of you know, last month we shared our strategy to position edithases as a leader in in vivo programs for gene editing.
Speaker 3: There are three underlying pillars of our strategy, which I will recap here. First, we have sharpened our discovery focus to in vivo administered genome editing medicines while continuing to develop Edit301 for severe sickle cell disease and transfusion dependent beta thalassemia or TDT.
Speaker 3: We continue to support our progress cell therapy programs, but are no longer discovering or developing standalone cell therapies. And we have divested our INK cell franchise to shoreline biosensis last month. We continue to support our INK cell therapy programs, but are no longer discovering or developing standalone cell therapies.
Speaker 3: Additionally, we terminated our AV-IRD program or platform and are seeking to divest those assets.
Speaker 3: Our sharpened discovery focus allowed us to concentrate our talent, which reduced our headcount by approximately 20% and extended our cash runway into 2025.
Speaker 3: Turning to our second strategic pillar, we are strengthening our discovery engine and technological capabilities. We have split our research division into separate technology and drone discovery groups enhancing the capabilities of each.
Speaker 3: Under our new target selection criteria, we will select therapeutic targets that will allow our genome-engaging approach to differentiate maximally from the current standard of care for serious diseases.
Speaker 3: Our goal here is to build a robust pipeline of assets that maximize the probability of technical, regulatory, and commercial success.
Speaker 3: Our new technology group under the leadership chief technology officer Bruce Eaton will focus on targeted delivery and Enhancing our gene editing toolbox to enable targeted gene repair
Speaker 3: Furthering innovation in our technology platform is a key component to achieving our goals via both internal clinical execution and external business development.
Speaker 3: Bruce is a biotech veteran who, in addition to his new role, would continue as chief business officer to spearhead our PD objectives. Within our drug discovery group, we have begun lead discovery work on in vivo therapeutic targets in hematopoietic stem cell or HSCs and other tissues.
Speaker 3: Our search for a new CSO who will head up our drug discovery group continues to progress, and I look forward to updating you on this search and our in vivo work in the future.
Speaker 3: Finally, our third strategic pillar is an increased and expanded approach to business development. Going forward, we will pursue the right combination of gene editing and targeted delivery tools through internal development and the in-licensing of complementary technologies in order to expedite our drug discovery and clinical execution objectives.
Speaker 3: In tandem, we will continue to leverage our IP portfolio to drive potential out licensing and partnership discussions.
Speaker 3: Moving to our development plans under the new strategy, as we shared last month, we are pursuing a lead-for-position in HSC therapeutics for hemoglobin opaties by taking three actions.
Speaker 3: First, we have reallocated investment resources to accelerate the clinical development of ETA-301 for the treatment of sickle cell and beta thalassemia.
Speaker 3: The positive initial data from my Ruby Phase 1-2 clinical study was a key driver of our decision to invest more heavily into Edit 301.
Speaker 3: Second, amphard. By leveraging our unique and differentiated approach of 301, we are investing to develop milder forms of patient pre-conditioning, as well as develop an in vivo approach for editing hematopoietic hemestined stem cell or HSEs, both of which should reduce the burden and improve the journey for patients who currently live with sickle cell diseases and TDD.
Speaker 3: Beyond hemoglobin optis, our discovery development efforts will be focused on in vivo at Minister Geno Mediging Medicine in other tissues.
Speaker 3: Turning to the clinic.
Speaker 3: Since we shared our strategy update last month, we have completed a review of the safety data from the sentinel patients of the Ruby trial for sickle cell disease and have begun parallel dosing of additional patients.
Speaker 3: We remain on track to provide an update on the Ruby clinical data mid-year and dose 20 total patients by year-end. An ambitious but certainly attainable goal.
Speaker 3: On Edit 301 for TDT, we remain on track to dose the first patient in our Edit File Phase 1, 2 trial and this quarter and provide an update from this trial by year-end.
Speaker 3: Taking this step back, I am confident in our strategy and we remain keenly focused on execution.
Speaker 3: We are building a pond of momentum from our clinical redact milestones during the fourth quarter and are recently announced deal with shoreline. We look forward to updating you on our progress and execution of our new strategy throughout the year. Now I will turn the call over to Bayesong, our chief medical officer.
Speaker 4: Thank you, Gilmore. Good morning, everyone.
Speaker 4: As Gail Moore mentioned, last December will present initial data from the Ruby trial of edis 3-1 for the first two patients with severe psychotherapy disease.
Speaker 4: That data for the first patient who had five months of follow-up should clinically significant improvement across all hematological parameters.
Speaker 4: These preliminary data suggest that editors have a product candidate that can potentially give robust clinical benefit to patients with severe sickle cell disease.
Speaker 4: and has the potential for clinical differentiation in the long term.
Speaker 4: Specifically, their patient had increased feed-of-and-globe infraction of 45.4% five months of shown that 27.2 meters remaining, and 42 kr Exercise session, teenage giraffe, and
Speaker 4: Above the 30% field of migrobing threshold where sick or cell patient may have no symptoms.
Speaker 4: We were also pleased to see that the patient total hemoglobin increased by more than 4g per desolidur to 16.4g per desolidur well into the normal range of male patients.
Speaker 4: And finally, the distribution of feed of migloving was highly pancellular.
Speaker 4: with 96% of sales.
Speaker 4: And the mean, capacicular fetal hemoglobin, or the fetal hemoglobin concentration, per red blood cells, increased to 13.8 ticogram per red cells.
Speaker 4: exceeding 10-figogram threshold considered clinically meaningful.
Speaker 4: As empirical evidence indicates, those levels will prevent that red blood cell from the nearby disease by calling change in a projects called compared to the risk of??, infection.
Speaker 4: As we have previously highlighted, Edits will want to utilize a unique magnet action.
Speaker 4: that added the promoted sequence of the gamma-glow-win genes to disrupt binding of BCL-11A suppressor.
Speaker 4: BIMMIC and the Nature Magnetism of hereditary persistence of fetal miglobe.
Speaker 4: The editing is done by a high-fability, highly specific engineered ASCAS-128 enzyme.
Speaker 4: In turn, this provides a high sustained level of feed home clothing, in a manner that can be independent of every through poetic stress.
Speaker 4: Residing in reduced cycling and the best occlusive event.
Speaker 4: your van skin, sick or sick or patient.
Speaker 4: and resolving anemia and transfusion dependence in better-theosemia patients.
Speaker 4: Since it allows in the initial data, we have completed our safety review of Sentinel patients from the Ruby trial for sickle cell disease.
Speaker 4: We are pleased to share that we continue to see a favorable safety profile.
Speaker 4: After completing sequential dosing of the first two patients, we have commenced parallel patient dosing, means that we can now dosing multiple patients simultaneously.
Speaker 4: And we remain on track to those 22-to-cyclic cell patients by year end.
Speaker 4: So our initial clinical data will very encouraging.
Speaker 4: consistent with pre-conquered data.
Speaker 4: We're confident we'll see replication of similar results in subsequent patients.
Speaker 4: We have said before that sickle cell disease is characterized by more than just the best or exclusive events.
Speaker 4: It can result in other severe symptoms such as the lemium fatigue, formalysis, stroke, and other organ damage.
Speaker 4: As we continue to monitor patients over time, we see potential for differentiation via longer term improvement in symptoms and more durable treatment.
Speaker 4: Since joining Eddie Best last year, I and the rest of management team has been focused on sharpening our clinical execution.
Speaker 4: We are very pleased with the momentum of edithry 1 in post, the Ruby and edithaic studies.
Speaker 4: Look forward to updating you and those trials progress.
Speaker 4: Now I will turn the call over to Michelle, our Chief Financial Officer, to review our financials.
Speaker 5: Thank you, Big Thou. Thank you, Morning, everyone. I'd like to refer you to our press meetings. It should be earlier today for a summary of our financial results in the fourth quarter in full year 2022.
Speaker 5: I'll take this opportunity to briefly review a few items.
Speaker 5: Our cash, cash equivalents and local security as of December 31, 437 million dollars compared to 620 million as of December 31, 2021.
Speaker 5: We expect our existing cash, cash equivalents, and multiple securities to fund our operating experiences in capital investments into 2025.
Speaker 5: Revenue for 2022 was approximately 20 million compared to 26 million and 2021. This decrease is mostly driven by a decrease in revenue recognized related to our collaboration agreement with BNF.
Speaker 5: GNA expenses decreased year-over-year from $76 million in 2021 to $71 million in 2022. The decrease was the net from an increase in strategy and later expenses offset by a decrease in legal and stock-based compensation.
Speaker 5: R&D expenses increased from 143 million in 2021 to 175 million in 2022.
Speaker 5: The $32 million increase was principally driven by manufacturing in clinical-related costs incurred to advanced editorial ones as well as a one-time charge incurred in connection with parking the enrollment in the bronze trial.
Speaker 5: Overall, edicass remains an extraordinary financial tradition.
Speaker 5: have the Guild Wars set our shop and discovery and development strategy and extend our cash word rate into 2025.
Speaker 5: This provides ample resources to support our continued trial of ed at 301, as well as enhancing our research efforts, human global monopathy, and other NVO discoveries.
Speaker 5: And with that, I will hear the call back to Gilmore.
Speaker 3: I'll hear the call back to Gilmore. Thank you, Michelle.
Speaker 3: In the almost eight months since I joined Edithas, the company has demonstrated two clinical POCs, one of which is the potential to be a competitive and a deep differentiated product. In addition, we have developed and shared our new strategy, reallocated resources to accelerate the clinical development of Edith 301.
Speaker 3: begun discovery of Indivo editing of HSCs and other tissues. Executed our goal to divest standalone celloprotherapy business with our shoreline deal.
Speaker 3: and are building up an already robust gene editing toolbox through the internal development, external business development of new and complementary technologies.
Speaker 3: And this is just the beginning. Looking into 2023, we look forward to executing on our strategy, continuing our transformation and sharing our progress with you.
Speaker 3: Other reminder, our strategic objectives for the year include hiring a new CSO with specific expertise aligned to our vision, refocusing our discovery group and fencing discovery of in vivo editing of HSCs and other tissues. On the development side, we plan to execute on the following in 2023.
Speaker 3: providing clinical updates from the Edit 301 Ruby study in mid-2023 and the end of 2023.
Speaker 3: which will include longer term data from the initial two patients that were dose last year as well as data from additional patients from the ongoing Ruby trial.
Speaker 3: In addition, dosing 20 total patients in our Edit 301 Ruby program by year end, dosing the first patient in the Edit 301 Editha trial for TTT this quarter, and finally providing early data in the Edit 301 Editha trial for TTT by year end.
Speaker 3: Thank you very much for your interest in Edithas and we are happy to answer questions now.
Speaker 1: If you would like to ask a question, please press star one on your telephone keypad. A confirmation tone will indicate your line is in the question queue. You may press star two if you would like to remove your question from the queue. And for a participant using speaker equipment, maybe necessary to pick up your hands up.
Speaker 1: before pressing the star keys. Our first question comes from June Lee with true with securities, please proceed.
Speaker 6: Hi, good morning. This is Mendeon for June . So I have two related questions. Do you expect for already half-take-as for ASCAS 12A and SLEC technology? And on ASCAS 12A...
Speaker 6: On the nature communication paper 2021, IDT mentioned that they have a patent on the mutations made for Castle of Ultra. So how similar or different is the AS Castle of A that you are using from?
Speaker 6: 2021, IDT mentioned that they have a patent on the mutations made for Castle of Ultra. So how similar or different is the AS Castle of A that you are using from Castle of Ultra?
Speaker 3: Thank you. Well, Mithy, I may have to ask you to pose your first question. I couldn't actually hear it.
Speaker 6: Oh, sorry. So do you expect or do you already have some other takers for ASK12A and slick technology?
Speaker 3: Oh, thank you, Matthew. Well, what I would say is that the most recent agreement or deal that we have done was with shoreline in which we gave them an exclusive license to our sleek knock-in technology for INK and their oncology derived or
Speaker 3: ASCAST12A and SLEEC as an example of licensing. And then with regard to your second question around the IP, we actually have a very comfortable estate that gives us the freedom to operate. And there are no interferences.
Speaker 3: or other disputes around IP for ASCAS 12-A.
Speaker 6: Thank you very much.
Speaker 1: Our next question is from Samantha Sevika with City. Please proceed.
Speaker 7: Good morning. Thanks for taking our questions. First question, I'm wondering what experience or expertise you're looking for specifically in your next CSO and if there's any expectation you could give us for when they could be appointed.
Speaker 3: Thanks very much, Samantha. We're looking for a number of different qualities and capabilities in our future CSO. We are looking for somebody who has experience in developing and bringing drugs into the clinic.
Speaker 3: We are looking for expertise and the ability to work with complex genomics groups. And we're also looking for somebody who has significant and important collaborative leadership capabilities for working with Bay Song.
Speaker 3: our chief medical officer with a Bruce Eaton, our chief technology officer and the executive team to help us in driving the selection of future therapeutic targets and target issues in which we will combine a number of elements.
Speaker 3: including the technology and the probability of technical success, as well as the probabilities of clinical regulatory and commercial success. And with regard to the timing, I'm happy with the progress we're making. We are talking to very interesting candidates.
Speaker 3: But the timing would really depend on making sure that we choose the best CSO for the organization. And what I will tell you is that I am very happy with the leadership that we have in our Discovery Group which is driving forward on our strategy.
Speaker 3: in the interim. Thank you.
Speaker 7: Thank you. Great. Thank you very much.
Speaker 3: Our next question is from DeGonha with Steefall. Please proceed. Good morning. Thanks for taking our questions. Clarification question and then one from me. On the clarification, so dosing 20 patients into Ruby by year end 23, do you think that's sufficient? Yes.
Speaker 8: to kind of move forward whether it's for regulatory filing or the pivotal trial. And then the question on B.D. when it comes to milder conditioning, just wanted to get your preliminary thoughts on where you think the direction is for potential differentiation for you guys.
Speaker 8: And if it pertains to CD117, then can you perhaps speak to what areas of differentiation you can actually contemplate here given the Jasper and Magenta updates?
Speaker 3: Yes, thanks very much, Decon. So with regard to the 20, we actually, that is actually going to generate a lot of very important clinical data. With regard to the regulatory needs, that will be a matter of discussion and agreement with the FDA and other regulators.
Speaker 3: of conditioning is that it actually significantly broadens the eligible patient population. Current conditioning is an extremely, as I say, severe.
Speaker 3: therapeutic intervention and many patients are precluded from using it just going to significant comorbidities which unfortunately result from the long-term complications of their disease. So mild conditioning per se doesn't have to be differentiated to actually substantially broadened.
Speaker 3: the patient population that could benefit from these therapies. And I think that's an important piece when you actually think about CD 117. With regard to other approaches, that is something that we are actively exploring and we'll be able to share more as we advance.
Speaker 3: on how we think that difference could be on the sort of the general and broad benefit to patients and eligibility through the development of milder conditioning.
Speaker 8: Great, thanks for the updates.
Speaker 1: Our next question is from Phil, they do with Count and Company, please proceed.
Speaker 8: Good morning, congrats on the progress and thanks for taking our questions. First, a cup on Edit 301. In terms of the mid-year update in the Ruby Trial, do you have a preliminary sense as to the number of patients in follow-up that will be in that look?
Speaker 9: And then on Edit 3.01 and TDT, two reminders is the design similar to Ruby and at the first two patients will be sequential dosing before moving the parallel dosing.
Speaker 3: Thanks Phil, I'm going to ask our chief medical officer, Bay Song, to answer those two questions.
Speaker 4: Thank you. Thank you for your question. We got your first question in the middle year of data release. We are, we will not share the number of patients this time, but we can share is that we, we will share the longer term data for the two patient dose last year and the additional data from multiple patient dose this year.
Speaker 4: And we have been not only those the sequential dosing the sentinel patients, but also started dosing the parallel dosing for the additional patients. And in addition to that, we have enrolled multiple patients and some of them already being with the emergency to be patients else committed to their workouts.
Speaker 4: 3D34 cells being edited and others being the process of a fluysis. So we are very confident and pleased with the progress of the Ruby Child progress.
Speaker 4: For your second question, can you review it again?
Speaker 9: Yeah, sorry in terms of 301 in and they'll see me
Speaker 9: Is the design the same as the Ruby trial and that the first two patients will be sequentially before you can move on to parallel dosing?
Speaker 4: Yeah, so we will have sendinel patients and we will share more details when we have more the data.
Speaker 9: Great, and then two follow up questions.
Speaker 9: In terms of the prior question, the mild preconditioning, do you have a sense when Editas could move that into the clinic when he could begin testing a milder preconditioning regimen? And then second, in terms of business development, you mentioned you were looking to bring complementary technologies.
Speaker 9: in-house, could you give us some sense of what complementary technologies you think you need?
Speaker 3: Yeah, thanks very much Phil. With regard to the modular reconditioning, we will actually talk more about that in the future, as in the appropriate time when we have some more details that we feel able to share with you.
Speaker 3: 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.
Speaker 3: 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.
Speaker 9: Perfect. Thanks for taking our questions.
Speaker 1: Our next question is from Rick Binkowski with Cantor Fitzgerald, please proceed.
Speaker 2: Thank you for taking the questions and congrats on all the progress here.
Speaker 9: So my first question is on the INVIVO pipeline. I know that the first two target indications are currently undisclosed, but it looks like the initial discovery efforts are going towards the INVIVO editing of STEM cells.
Speaker 9: Could you highlight the reasons why you showed this as the initial area of focus and why it could be advantageous to target these cells rather than an organ-like deliver or methods of delivering gene editors are more established?
Speaker 9: And then for my second question for the goal of dosing 20 patients by year end in the sickle cell trial. Are there any important limitations in manufacturing capacity or as available that would limit the number of patients that you can dose in parallel? And you know, 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.
Speaker 3: which we have not disclosed. The reason that we actually talked about HSCs is that, you know, are several. One of which is that there's a lines with a key focus of our active pipeline. It builds on the success of our Edit 301. And essentially, we have somewhat simplified.
Speaker 3: the calculus by having a human validated enzyme or effector enzyme in ASCAS 12A and a human validated target in the HG-1-2 promoter which enables us to really focus.
Speaker 3: largely or predominantly on the targets of delivery. I will say, however, that we are actually looking at other tissues, but we haven't disclosed those yet.
Speaker 3: Turning to your second questions with regards to limitations, there are, we have actually substantially invested and reallocated capital with our new strategy to CMC and do not have limitations there to both.
Speaker 3: editing and supporting parallel dosing. But indeed, you know, based on could probably tell you more about the enthusiasm in the community following our day's disclosure and our plans for the year. Yeah, yes, sure. Yes, Rick, yes, that's a very good question. So we, as Gimma mentioned, we really be fun out our...
Speaker 4: We really see a strong momentum over the last several years. So we're very pleased to that.
Speaker 4: to see the number of patients being enrolled, being in screening as well as the prospective patients we have in there.
Speaker 4: So we are very confident we'll be able to achieve the goal to 20 patients by your end.
Speaker 3: I think when you said previous years, I think you mean previous months and weeks. Last several months, yes. Thank you. And a lot of enthusiasm and excitement from patients and investigators when we disclose the initial 301 data in December . Thanks very much. Thank you very much.
Speaker 1: Great. Thank you. Our next question is from Joel Beady with Baird. Please proceed.
Speaker 10: Thank you so much for taking the questions that Jack Allen, Don, and for Joel. We're hoping you can revot an update as it relates to the IP Dynamics surrounding CRISPR Cat 9 and your IP there in light of the February 22 decision by the USPTO. I ask as your competitor, CRISPR and Rivertex, is looking to commercialize CTX-0s Air 1 users.
Speaker 10: cast 9 based therapy for sickle cell and beta-thalin in late 23 early 24. And I was wondering if you had any thoughts as it relates to the time course of potential, I guess, settlements or any litigation surrounding IP of that cast 9 portfolio.
Speaker 3: Thank you so much. Thanks very much, Jack, for the question. And obviously, we were very happy that the Broad's IP was upheld by the P-Tab last February . I think the key thing is that this is a very exciting time and we're delighted for patients. This is an exciting time because we're looking potentially at the first approval for a CRISPR-based medicine for patients.
Speaker 3: Thank you.
Speaker 1: Our next question is from Jay Olson with Oppenheimer, please proceed.
Speaker 9: Oh, hey, thank you for the update and thanks for taking a look.
Speaker 10: questions. Can you talk about any feedback that you're receiving from your independent data monitoring community and their latest review of the Ruby trial?
Speaker 11: And then...
Speaker 10: The second question, as a result of your portfolio re-cryroreterization process.
Speaker 10: Are there any implications for operating expenses or your capital allocation strategy this year? Thank you.
Speaker 4: Thank you very much. Based on, do you want to talk about the IDMC feedback and the release for parallel dosing? Yeah, sure. Thanks, Jair, for the question. Yes, the IDMC had reviewed the data from the two Sentinel patients and they agreed for about the fifth attempt.
Speaker 4: continued and dozing and parallel dozing the patient.
Speaker 3: Thanks very much for your song. And then with regard to the implications for off-pecks and capital allocation, let us pass that to Michelle. Great. Hi Jay. So with the refocus strategy, we were able to extend our cash runway into 2025.
Speaker 5: We don't give out guidance on expenses, but obviously you can back into that given that our cash balance was $137 million of years of the year. And we'll continue to invest in editorial one, both of the clinical trials, continue to invest in CMC.
Speaker 5: And then obviously in our advanced technology and further in the platform.
Speaker 9: Great. Thank you very much. Thanks very much, Misha.
Speaker 3: Thank you very much. Thanks very much, Misha. Thanks.
Speaker 1: Our next question is from Brian Chang with JP Morgan. Please proceed.
Speaker 2: Hey guys, thanks for taking my call this morning. Kim, what it looks like your collaboration with Bristol is going fairly well. I'm just curious if you can provide us some color on the collaboration. You know, what were the factors that prompted Bristol to open to more programs?
Speaker 2: Any color on the next step and how wide are the indications that the 10 programs are covering?
Speaker 2: steps and how wide are the indications that the 10 programs are covering. Thank you.
Speaker 3: Thanks very much, Brian . You know, we're actually very happy with the BMS collaboration. I think one of the things that is driving options is that I believe that we have been providing very high quality packages around the target nominations. The...
Speaker 3: Our collaboration is focused on alpha-basit T cells for the treatment of oncology and covers a rather broad sway of possibilities, which we haven't actually disclosed publicly, but I think you can infer from the nature of the agreement.
Speaker 3: the target cell types and the oncology where it is likely to be. I think more details will be forthcoming as the MS advances the programs.
Speaker 3: Thanks Brian . Thank you. Good work.
Speaker 1: Our next question is from Luca. Is that with RBC capital markets, please proceed?
Speaker 12: Oh great, thanks so much for staying on my question. Congrats on all the progress. I have two quick ones. Maybe circling back on a prior question based on if I may. I think you've spoken in the past about the Ruby trial, potentially becoming registrational. Obviously you're now flagging that you'll have 20 patients by year end.
Speaker 12: How many patients and what is the minimum follow-up that you think is required to actually have a Registrational package, again any call there, we much appreciated, and then we give more for you on LCA 10 and IRD more broadly. Can just give us an update on what is strategic option that you're exploring in the moment.
Speaker 4: won't be an ideal partner and ideal deal there. So thanks so much. Thanks, Luca, for your question. So just wanted to clarify, we have ambitious but a ton of goal to those total of 20 patients by year end. That is a separate form of the registration of package. As Gilmore mentioned a bit earlier, that for the registration of package, a number of people are going to be in the
Speaker 3: potency matrix or assay matrix that all the patients do in Ruby will actually provide data that would be part of marking application. With regard to your second question and strategic options for our IRD divestiture, we are actually exploring potential divestitures.
Speaker 3: An ideal partner would be a sponsor interested in rare ocular diseases and potentially with a particular interest in AV delivery. But that's not a complete restriction. But an ATT expertise in rare retinal disorders.
Speaker 3: and the development would be an ideal partner for us. And we look forward to being able to update you in the future on the progress we make.
Speaker 12: Thanks so much.
Speaker 1: Our next question is from Greg Harrison with Bank of America. Please proceed.
Speaker 13: Hey, good morning. Thanks for taking the questions. First, I just wanted to ask on expectations for the sickle cell data.
Speaker 13: I know it's been discussed previously, but just wanted to get your latest thoughts on what you need to show to really have a competitive profile here in sickle cell. And then on another note, what else needs to happen with the reorganization and how close are you to being complete with that?
Speaker 3: on Feetalhema Globen and in the resolution of Anemia. And we are looking forward and confident we can see replication and supple and patients for that data set and look forward to sharing updates in the middle and at the end of this year.
Speaker 3: And then with regard to reorganization, we have largely completed that reorganization. I think the critical element of the reorganization that is outstanding is the hiring of our Chief Scientific Officer. I have already talked a little bit about the characteristics, traits, qualities we're looking for.
Speaker 1: Great, that's helpful. Thanks for taking the questions. Our next question is from Ian and Zoo with Wells Fargo. Please proceed.
Speaker 9: Hi, and thanks for the questions. Have you dosed the third patient in the sickle cell trial yet? That's the first question. And at what point along the date of generation process from the sickle cell trial? Well...
Speaker 3: Do you plan to reach out and have a discussion with the regulator about the path forward for edith 301? And lastly, is the 301's manufacturing process?
Speaker 9: carried out with a commercial grade process.
Speaker 14: And IE would some kind of bridging needed if it is not that kind of a process currently.
Speaker 4: Thanks very much, Janan. Face-on, if you don't just sort of recapitulate the updates. Yeah, yeah. So thanks for your question. And I get the first two questions. I'll let you know you answered the question about the manufacturing in there, too. So yes, and then we actually, we did dose the third patient and started as we mentioned that.
Speaker 3: and we'll share more data when times appropriate. Thanks very much, Bay Song. With regard to our 3-1 process, the good news is that the certainly on the potency matrix area, we have actually...
Speaker 3: being very successful and ensuring that the patients that we are currently dosing in Ruby will be able to support a Marketing application with regard to the refinements and a great final commercial process that would be something that we will update you Obviously at a time the appropriate and obviously we include
Speaker 14: You know more details. Great. Thanks for the color.
Speaker 1: Our next question is from Gina Wang with Barclays. Please proceed.
Speaker 7: Thank you. I have three quick questions. The first one is regarding the shoreline deal. Can you remind us the deal term and the economics? And when you expect the transaction to be closed? Does your cash guidance take into consideration of an impact from the deal? That's the first question. Thank you.
Speaker 3: Oh, thanks, Jean, oh, you're very good. You're going to keep them. That's great, so we don't have to track them all. So with regard to the shoreline deal, we didn't actually disclose the numbers in the economics. We did receive an upfront. We are eligible for future development and commercial milestones and royalty payments. Thank you.
Speaker 3: with the advance of the assets, indeed that those impact in the financials is actually included or has been considered. So that's, I think, the first question.
Speaker 7: Okay. Have you already received the payment or you will receive after transaction closed?
Speaker 3: We've received the payment, Gina. Yeah, actually, I actually forgot to answer your part of your question, Gina, which is with the clothes that we have clothes to do, we have received the payment.
Speaker 7: The payment was received in 2023. I see. So the cash reported already incorporated that.
Speaker 5: The cash report at the end of the year did not yet include that.
Speaker 5: Okay, the guide. That will be in our re- that will be in our re-forecast of our cash roadway.
Speaker 7: Okay, sounds good. And the second question very quick, just, I know you are committed to present the data, 301 RubyTrial mid-year. What about the end of 2023, especially like the ash, will you also provide some update there and thoughts on the timing and also number of patients?
Speaker 3: Yeah, thanks Gina. I'm going to ask Bay Song to answer that question, but you know confirm that we are planning to present update both the middle of this year and at the end of this year with Bay Song.
Speaker 4: Thanks, Gina, for the question. Yes, we were planning to have two days of release for Ruby study when is the mid of the year and when is the end of the year. In terms of the specific forum, we will share when we have more information. And we are not to share the number of patients at this point, but I just mentioned earlier.
Speaker 4: we really have a strong momentum for the Ruby study as well and it's a study. And we expect to have multiple patient data for the data release.
Speaker 7: Okay, and then lastly very quick in vivo programs beyond HSC, what would be your top organ to explore?
Speaker 3: Thanks very much, Gina. We actually have taken the approach that we will use as sort of a balance of multiple factors in selecting our targets, our therapeutic targets, and their core forergo the target tissues, and that would be a balance of...
Speaker 3: the state of the technology, the probability of clinical regulatory and commercial success, and we look forward to sharing more specifics on that in the future.
Speaker 15: Thank you.
Speaker 15: Thank you. Thanks very much.
Speaker 1: Our next question is from Rich Law with Credit Svists. Please proceed.
Speaker 3: Good morning and thanks for taking my question. As you focus on in vivo development, is there any we knew interest in using CRISPR Cas9 in addition to Cas 12 for internal product development? Since the Cas9 technologies arguably more mature from the development progress by other companies. And then I have a follow-up question to that.
Speaker 3: Thanks very much, Rich. You know, I have to say that our pipeline currently is focused on ASCAST12A. We believe that the ASCAST12 at Enzheim has some significant advantages. We actually also believe that now we have used it in the clinic. We also believe that the ASCAST12A has some significant advantages.
Speaker 3: And actually, as Bayesang has said, we've already edited multiple patients in addition to those already dozed. So we actually are building a clinical experience with ASCAS 12A. And from the point he was advantageous, we believe that and actually have data supporting that position that it has a higher efficiency.
Speaker 3: and essentially higher fidelity with much less off-target editing, so as substantial advantages for moving forward.
Speaker 3: Okay. A follow-up question to that is that is there any performance requirement in the licensing agreement that you have with the growth that you have to continuously perform proxie development with the cast night technology?
Speaker 3: I'm not going to go into details, but obviously, like any agreement there are...
Speaker 3: conditions or requirements to execute and ours are a mixture around both on our business development and execution.
Speaker 16: I.
Speaker 15: Great. Thank you.
Speaker 1: Our next question is from Medu, Kumar with Goldman Sachs. Please proceed.
Speaker 12: Hey, thanks for taking our question. This is Rob on from Adoo. Two quick ones. What venue should we expect the mid-23-301 update? It's like medical meeting or company release. And then how do you think about the regulatory path in NVivo, CRISPR and the US, given the headwinds we've seen by some of your peers? Thank you.
Speaker 4: Based on, do you take the first question, do I take a second? Sure, yes. So we plan to release the Ruby data mid of the year as well as the end of the year. The specific venue we have not decided will share the latest time.
Speaker 3: Thanks very much, Jason. And then with regard to the regulatory path for In vivo, CRISPR, what I would actually say, I'm not going to comment on others' experiences, the regulators. I would say that one of the things that drew me to Edithas was the substantial investment and death of expertise built.
Speaker 3: in both its research analytics as well as its CMC analytics from the point of view of certainly managing risk, et cetera. So I actually feel very good about where we are from that point of view. And obviously as we move forward, we will engage with regulators.
Thanks for taking out questions. This is Chris on for Matthew. We have two questions. One is about the SCD filing package. You mentioned a few times that you need to talk to the regulators about that, but do you think it's possible that you could be in a position to file in 2024?
And then the second question is about the BMS collaboration. Is it possible to see an update in 2023 and how should we think about any of those programs progressing into the clinic? Thank you.
Thanks very much. I think let me take both questions regarding the finding package. Certainly I admire your aggressive posture there. I think any question around timing of filings and what is required is really a matter of negotiation with the agencies.
And that's only something that we will know as we progress or negotiations with them. With regards to the BMS collaboration, we are happy with the collaboration.
The MS has actually received a lot of nice data from us and actually nominated a number of targets. We hope that that will continue to progress. But with regard to the specifics of advancing towards the clinic, that really is driven by.
and the announcements are driven by BMS. They say we're very excited by the collaboration and are delighted to see the progress.
Thank you very much. Our final question is from Lisa Beko with Evercore ISI. Please proceed.
Hi, all my questions been answered, but thanks for taking me on thick.
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Thanks Lisa.
Thank you. This will conclude today's conference. You may disconnect your lines at this time and thank you for your participation.
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