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

Editas Medicine, Inc. (NASDAQ:EDIT) Q4 2022 Results Conference Call February 22, 2023 8:00 AM ET

Company Participants

Ron Moldaver - Investor Relations

Gilmore O’Neill - Chief Executive Officer

Michelle Robertson - Chief Financial Officer

Baisong Mei - Chief Medical Officer

Conference Call Participants

Samantha Semenkow - Citi

Dae Gon Ha - Stifel

Phil Nadeau - Cowen & Company

Rick Bienkowski - Cantor Fitzgerald

Jay Olson - Oppenheimer

Brian Cheng - JP Morgan

Luca Issi - RBC Capital Markets

Greg Harrison - Bank of America

Yanan Zhu - Wells Fargo

Gena Wang - Barclays

Rich Law - Credit Suisse

Liisa Bayko - Evercore ISI

Operator

Good morning and welcome to Editas Medicine’s Fourth Quarter and Full-year 2022 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 Ron Moldaver, Investor Relations for Editas Medicine.

Ron Moldaver

Thank you, Sherry. Good morning, everyone and welcome to our fourth quarter and full-year 2022 conference call. Earlier this morning, we issued a press release providing our financial results and recent corporate update. 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.

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 statement, even if our views change.

Now, I will turn the call over to our CEO, Gilmore O’Neill.

Gilmore O’Neill

Thank you, Ron and good morning, everyone. 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.

Last year was a landmark year in Editas Medicine’s history as our team demonstrated two chemical proof-of-concepts 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 EDIT-101 AV delivered Cas9 therapeutic LCA-10 an inherited retinal disease. However, due to the limited addressable population we made the tough decision to pause patient enrolment in our BRILLIANCE trial. In

December we shared very encouraging initial data from the RUBY Phase 1/2 clinical study of our ex-vivo autologous EDIT-301 therapy in severe sickle cell disease. The readout provided human proof-of-concept demonstrating that EDIT-301 could safely drive expression of fetal hemoglobin to clinically meaningful levels and correct anemia in sickle cell disease patients, and the study continues to progress.

As we move into 2023, we will build on these successes as we continue to drive towards our goal of delivering life-saving medicines to patients with previously untreatable or undertreated diseases. As many of you know, last month, we shared our strategy to position Editas as a leader in in-vivo program for gene editing.

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 EDIT-301 for severe sickle cell disease and transfusion Dependent Beta Thalassemia or TDT.

We continue to support our partnered cell therapy programs, but are no longer discovering or developing standalone cell therapies, and we have divested our iNK Cell Franchise to Shoreline Biosciences last month.

Additionally, we terminated our AAV IRD program or platform and are seeking to divest those assets. Our sharpened discovery focus allowed us to concentrate our talent, which reduced our headcount by approximately 20% and extended our cash runway into 2025.

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 drug discovery groups, enhancing the capabilities of each.

Under our new target selection criteria, we will select therapeutic targets that will allow our genome managing approach to differentiate maximally from the current standard of care for serious diseases. Our goal here is to build a robust pipeline of assets that maximize the probability of technical, regulatory and commercial success.

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. Furthering innovation in our technology platform is a key component to achieving our goals via both internal clinical execution and external business development. Bruce is a biotech veteran who, in addition to his new role would continue as Chief Business Officer to spearhead RPD objectives.

Within our drug discovery group, we have begun lead discovery work on in-vivo therapeutic targets in hematopoietic stem cells or HSCs and other tissues. 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.

Finally, our third strategic pillar is an increase in 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. In tandem, we will continue to leverage our IP portfolio to drive potential out licensing and partnership discussions.

Moving to our development plans under the new strategy, as we shared last month, we are pursuing a leadership position in HSC therapeutics for hemoglobinopathies by taking three actions.

First, we have reallocated investment resources to accelerate the clinical development of EDIT-301 for the treatment of sickle cell and beta thalassemia. The positive initial data from our RUBY Phase 1/2 clinical study was a key driver of our decision to invest more heavily into EDIT-301.

Second and third, by leveraging our unique and differentiated approach of 301, we are investing to develop milder forms of patient preconditioning as well as develop an in-vivo approach for editing hematopoietic stem cell cells or HSC, both of which should reduce the burden and improve the journey for patients who currently live with sickle cell diseases and TDT. Beyond hemoglobinopathies our discovery and development efforts will be focused on in-vivo administered genome editing medicines in other tissues.

Turning to the clinic. Since, we shared our strategy update last month, we have completed 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.

We remain on track to provide an update on the RUBY clinical data at mid-year and dose 20 total patients by year-end, an ambitious but certainly attainable goal. On EDIT-301 for TDT, we remain on track to dose the first patient in our EDITHAL Phase 1/2 trial this quarter and provide an update from this trial by year-end.

Taking a step back, I’m confident in our strategy, and we remained keenly focused on execution. We are building upon the momentum from our clinical redact milestones during the fourth quarter and our 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 Baisong, our Chief Medical Officer.

Baisong Mei

Thank you, Gilmore. Good morning, everyone. As Gilmore mentioned, last December, we presented initial data from the RUBY trial of EDIT-301. For the first two patients with a severe sickle cell disease, that data for the first patient who had a five-months of follow-up showed clinically significant improvement across all hematological parameters.

These preliminary data suggests that Editas has a product candidate that can potentially give robust clinical benefit to patients with severe sickle cell disease, and has the potential for clinical differentiation in the long-term.

Specifically, that patient has increased fetal hemoglobin infraction of 45.4%, five-months of EBIT-301 infusion. Above the 30% fetal hemoglobin threshold where sickle cell patient may have no symptoms. We were also pleased to see that the patient’s total hemoglobin increase by more than four gram per deciliter to 16.4 gram per deciliter well into the normal range of male patients.

And finally, the distribution of fetal hemoglobin was highly pan cellular with 96% F-cells and the Mean Corpuscular fetal hemoglobin or the fetal hemoglobin concentration per red blood cells increased to 13.8 pg gram per red cells, exceeding 10 pg gram threshold considered clinically meaningful as empirical evidence indicates those level will prevent that red blood cell from sickle.

As we have previously highlighted, EDIT-301 utilize a unique mechanism of action that edit the promoted sequence of the gamma-globin genes to disrupt binding of BCL11A suppressor, mimicking the nature mechanism of hereditary persistence of fetal hemoglobin. The editing is done by a high fidelity, highly specific engineered AsCas12a enzyme.

In turn, this provides a high sustained level of fetal hemoglobin in a manner that can be independent of every through periodic stress, residing in reduced, sickle, and the vessel occlusive events in sickle cell patients, and resolving anemia and transfusion dependence in beta thalassemia patients.

Since launching the initial data, we have completed our safety review of sentinel patients from the RUBY trial for sickle cell disease. We are pleased to share that we continue to see a favorable safety profile. After completing sequential dosing of the first two patients, we have commenced parallel patient dosing, means that we can now dosing multiple patients simultaneously and we remain on track to dose 22 sickle cell patients by year-end.

Our initial clinical data were very encouraging, consistent with preclinical data. We are confident we will see replication of similar results in subsequent patients. We have said before that sickle cell disease is categorized by more than just vessel occlusive events.

It can result in other severe symptoms such as anemia, fatigue, hemolysis, stroke and other organ damage. As we continue to monitor patients over time, we see potential for differentiation via longer term improvement in symptoms and more durable treatment.

Since joining Editas last year, I and the rest of management team has been focused on sharpening our clinical execution. We are very pleased with the momentum of EDIT-301 in both the RUBY and EDITHAL studies. Look forward to updating you as those trials progress.

Now I will turn the call over to Michelle, our Chief Financial Officer, to review our financials.

Michelle Robertson

Thank you Baisong, and good morning, everyone. I would like to refer you to our press release issued earlier today for a summary of our financial results for the fourth quarter and full-year 2022. I will take this opportunity to briefly review a few items.

Our cash, cash equivalent and marketable securities as of December 31^st were $437 million compared to 620 million as of December 31, 2021. We expect our existing cash, cash equivalent and marketable securities to fund our operating expenses and capital investment into 2025.

Revenue for 2022 is approximately 20 million compared to 26 million in 2021. This decrease is mostly driven by a decrease in revenue recognized related to our collaboration agreement with BMF.

G&A expenses decreased year-over-year from 76 million in 2021 to 71 million in 2022. The decrease was the net of an increase in strategy and legal expenses offset by a decrease in legal and stock based compensation.

R&D expenses increased from 143 million in 2021 to 175 million in 2022. The $32 million increase was principally driven by manufacturing and clinical related costs incurred to advance EDIT-301 as well as a one-time charge incurred in connection with pausing enrollment in the BRILLIANCE trial. Overall Editas remains in a strong financial position.

As Gilmore said, our shop and discovery and development strategy extends our cash runway into 2025. This provides ample resources to support our continued trials of EDIT-301 as well as advancing our research ethics and Hemoglobinopathy and other in-vivo discovery.

And with that, I will hand the call back to Gilmore.

Gilmore O’Neill

Thank you, Michelle. In the almost eight-months since I joined Editas, the Company has demonstrated two clinical POCs, one of which has 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 EDIT-301. Begun discovery of in-vivo editing of HSCs and other tissues, executed our goal, divest standalone seller for therapy business with our shoreline deal and our building up an already robust gene editing toolbox through the internal development, external business development of new and complementary technologies 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. As a reminder, our strategic objectives for the year include hiring a new CSO with specific expertise aligned to our vision, refocusing our discovery group, advancing discovery on in-vivo, editing of HSC and other tissues.

On the development side, we plan to execute on the following in 2023, providing clinical updates from the EDIT-301 RUBY study in mid-2023 and the end of 2023, which will include longer term data from the initial two patients that were dosed last year, as well as data from additional patients from the ongoing RUBY trial.

In addition, dosing 20 total patients in our EDIT-301 RUBY program by year-end, dosing the first patient in the EDIT-301 EDIT trial for TDT this quarter, and finally providing early data in the EDIT-301 EDITHAL trial for TDT by year-end.

Thank you very much for your interest in Editas, and we are happy to answer questions now. Thank you.

Question-and-Answer Session

Operator

[Operator Instructions] Our first question comes from Joon Lee with Truist Securities. Please proceed.

Unidentified Analyst

Hi good morning. This is [Mattie] (Ph) on for Joon. So I have two related questions. Do you expect or already have takers for AsCas12a and slick technology and on AsCas12a, on the nature of communication paper 2021, IDT mentioned that they have a patent on the mutations made for Cas12a Ultra. So, how similar or different is the AsCas12a that you are using from Cas12a Ultra? Thank you.

Gilmore O’Neill

Well, Matthew, I may have to ask you to an pose your first question. I couldn’t actually hear it.

Unidentified Analyst

Sorry. So, do you expect or you already have some other takers for AsCas12a and slick technology?

Gilmore O’Neill

Thank you, Mattie. 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 driven iMac platform, and an additional non-exclusive license for AsCas12a. That was in addition to obviously selling the EDIT-202 asset. So I think that is where we are on AsCas12a and sleek as an example of licensing.

And then with regard to your second question around AIP, we actually have a very comfortable estate that gives us the freedom to operate. And there are no interferences or other disputes around IP for AsCas12a.

Unidentified Analyst

Thank you very much.

Operator

Our next question is from Samantha Semenkow with Citi. Please proceed.

Samantha Semenkow

Good morning, thanks for taking our questions. First question, I’m wondering what experience or expertise you are looking for specifically in your next CSO, and if there is any expectation you could give us for when they could be appointed.

Gilmore O’Neill

Thanks very much, Samantha. We are 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. We are looking for expertise, and the ability to work with complex genomics groups.

And we are actually also looking for somebody who has significant important collaborative leadership capabilities for working with Baisong, our Chief Medical Officer with Bruce Eaton, our Chief Technology Officer, and the executive team to help us in driving the selection of future therapeutic targets and target tissues in which we will combine a number of elements, 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 are making. We are talking to very interesting candidates. But the timing will really depend on making sure that we choose the best CSO for the organization. And what I will tell you is that I’m very happy with the leadership that we have in our discovery group which is driving forward on our strategy in the interim. Thank you.

Samantha Semenkow

Okay. Thank you very much.

Operator

Our next question is from Dae Gon Ha with Stifel. Please proceed.

Dae Gon Ha

Hey 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 2023. Do you think that is sufficient to kind of move forward whether it is for regulatory filing or the pivotal trial?

And then the question on BD, 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. And if it pertains to CD-117, then can you perhaps speak to what areas of differentiation you can actually contemplate here, given the Jasper and Magenta updates? Thanks.

Gilmore O’Neill

Yes, thanks very much Dae Gon. So with regard to the 20, we actually be - 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 regulatory agencies. And that is just something that we are planning over the coming year.

Now with regard to milder conditioning and differentiation. I think, there are a number of ways of looking at that. I think the key thing about milder conditioning is that it actually significantly broadens the eligible patient population.

Current conditioning is an extremely how should I say, severe therapeutic intervention. And many patients are precluded from using it, just going to significant co-morbidities, 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 broaden the patient population that could benefit from these therapies. And I think, that is 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 will be able to share more as we advance on how we think that differentiates beyond the sort of the general and broad benefit to patients eligibility through the development of milder conditioning.

Dae Gon Ha

Great. Thanks for the updates.

Operator

Our next question is from Phil Nadeau with Cowen & Company. Please proceed.

Phil Nadeau

Good morning. Congrats on the progress and thanks for taking our questions. First a couple 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 work? And then on EDIT-301 in TDT, can you remind us, the design similar to RUBY and that the first two patients will be sequential dosing before moving to parallel dosing?

Gilmore O’Neill

Thanks Phil. I’m going to ask our Chief Medical Officer Baisong to answer those two questions.

Baisong Mei

Thank you. Thank you, Phil for your question. Regarding your first question in the middle year of data release, we will not share the number patient this time, but what we can share is that we will share the longer term data for the two patient dose last year and the additional data from multiple patient dosed this year.

And we have been not only dosed the sequential dosing the sentinel patient, 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 been - CD34 cell is being edited and others be in the process of recess. So we are very confident and pleased with the progress of the RUBY trial progress.

For your second question, can you repeat again?

Phil Nadeau

Yes, sorry. In terms of 301 and thalassemia is the design the same as the RUBY trial and that the first two patients will be dosed sequentially before you can move on to parallel dosing?

Baisong Mei

Yes. So we will have sentinel patients and we will share more details when we have more of the data.

Phil Nadeau

Great. And then two follow-up questions in terms of the prior question, the mild preconditioning, do you have a sense when Editas could move that into the clinic when you could begin testing a milder preconditioning regimen?

And then second, in terms of business development, you mentioned you were looking to bring complementary technologies, in-house. Could you give us some sense of what complementary technologies you think you need? Thank you.

Gilmore O’Neill

Yes, thanks very much, Phil. With regard to the milder preconditioning, we will actually talk more about that in the future at an appropriate time when we have some more details that we feel able to share with you.

With regard to the complementary technologies, just in general, we are looking across the two elements of advanced technology that will be important to driving our in-vivo focus, and those around targeted delivery and obviously additional targeted editing approaches. And again, we will be able to share more details about that in the future.

Phil Nadeau

Perfect. Thanks for taking our questions.

Gilmore O’Neill

Thanks very much, Phil.

Operator

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

Rick Bienkowski

Hi everyone. Good morning. Thanks for taking the questions and congrats on all the progress here. So my first question is on the in-vivo pipeline. I know that the first two target indications are currently undisclosed, but it looks like the initial discovery efforts are going towards the in-vivo editing of stem cells. Could you highlight the reasons why you chose this as the initial area of focus and why it could be advantageous to target these cells rather than an organ like the liver where methods of delivering gene editors are more established?

And then for my second question, for the goal of dosing 20 patients by year-end in a sickle cell trial. Are there any important limitations in manufacturing capacity or bed available that would limit the number of patients that you can dose in parallel and I guess what I’m really trying to understand here is if patient dosing is likely to be evenly distributed over the year, or if it could be weighted more towards the back half of the year?

Gilmore O’Neill

Thanks very much Rick for those questions. Let me turn to the in-vivo pipeline question, while we did talk about in-vivo HSCs, indeed we are looking at other tissues, which we have not disclosed. The reason that we actually talked about HSCs is that our several one of which is that it aligns with a key focus of our active pipeline.

It builds on the success of our EDIT-301 and essentially we have somewhat simplified the calculus by having a human validated enzyme or a factor enzyme in AsCas12a and a humanly validated target in the HPPG 12 promoter, which enables us to really focus largely or predominantly on the target delivery. I will say, however, that we are actually are looking at other tissues but we haven’t disclosed those yet.

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

Baisong Mei

Yes, sure. Rick, yes, that is a very good question. So, as Gilmore mentioned, we really beefing up our CMC capacity as we discussed before. We actually have a strong CMC team internally be able to handle all those - handle the internal CMC manufacturing capacities and working with partners.

In terms of the - we have patient recruitment, we really see a strong momentum over the last several years. So we are very pleased to see the number of patients being enrolled, in screening as well as the prospective patients we have in there. So, we are very confident we will be able to achieve the goal 20 patient by year-end.

Gilmore O’Neill

I think when you said previous years, I think you meant previous months and weeks.

Baisong Mei

Last several months as well, yes sir.

Gilmore O’Neill

Yes. Thank you. And a lot of enthusiasm and excitement from patients and investigators when we disclosed the initial 301 data in December. Thanks very much.

Rick Bienkowski

Great, thank you.

Operator

Our next question is from Joel Beatty with Baird. Please proceed.

Unidentified Analyst

Hi thank you so much for taking the questions, it is [Jack Allen] dialing-in and for Joel. We were hoping you can provide an update as it relates to the IP dynamics surrounding CRISPR/Cas9, and your IP there in light of the February 2022 decision by the USPTO. I ask as your competitor, CRISPR and Vertex is looking to commercialize CTX001 as aCAS9 based therapy for sickle cell and beta thalassemia in late 2023, early 2024. 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 CAS9 portfolio? Thank you so much.

Gilmore O’Neill

Thanks very much, Jack for the question. And obviously, we were very happy that the broads IP was upheld by the PTAB last February. I think, the key thing is that this is a very exciting time and we are delighted for patients.

This is an exciting time, because we are looking potentially at the first approval for a CRISPR based medicine for patients with very serious diseases. Obviously, we look to a negotiated agreement to enable our -- the continuing advance of the technology, and we will be updating you on the details of that an appropriate time.

Unidentified Analyst

Thanks so much.

Gilmore O’Neill

Thank you.

Operator

Our next question is from Jay Olson with Oppenheimer. Please proceed.

Jay Olson

Hey thank you for the update and thanks for taking the questions. Can you talk about any feedback that you received from your independent data monitoring community and their latest review of the RUBY trial?

And then a second question, as a result of your portfolio reprioritization process, are there any implications for operating expenses or your capital allocation strategy this year? Thank you.

Gilmore O’Neill

Thank you very much. Baisong, do you want to talk about the IDMC feedback and the release for parallel dosing?

Baisong Mei

Yes, sure. Thanks, Jay, for the question. Yes. The IDMC had reviewed the data from the two Sentinel patients, and they agreed for continued and dosing and parallel dosing of the patients.

Gilmore O’Neill

Thanks very much, Baisong. And then with regard to the implications for OPEX and capital allocation, I’m going to pass that to Michelle.

Michelle Robertson

Great. Hi, Jay. So with the refocus strategy, we were able to extend our cash runway into 2025. We don’t give out guidance on expenses, but obviously you can back into that. Given that our cash balance was 437 million at the end of the year, and we will continue to invest in EDIT-301, both of the clinical trials continue to invest in CMC, and then obviously in our advanced technology and further in-vivo platform.

Jay Olson

Great, thank you very much. Thanks very much Michelle. Thanks.

Operator

Our next question is from Brian Cheng with JP Morgan. Please proceed.

Brian Cheng

Hey guys thanks for taking my call this morning. Gilmore, 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. What were the factors that prompted, Bristol to open to more programs, any color on the next steps and how wide are the indications that the 10 programs are covering? Thank you.

Gilmore O’Neill

Thanks very much, Brian. We are actually very happy with the BMS collaboration. I think, one of the things that is driving opt-ins is that I believe that we have been providing very high-quality packages around the target nominations.

The program or collaboration is focused on alpha based 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, the target cell types and the oncology where there is likely to be. And I think more details will be forthcoming, as BMS advances the programs. Thanks Brian.

Brian Cheng

Thank you Gilmore.

Operator

Our next question is from Luca Issi with RBC Capital Markets. Please proceed.

Luca Issi

Great. Thanks so much for taking 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 are now flagging that you’ll have 20 patients by year-end. How many patients and what is the minimum follow-up that you think is required to actually have a registrational package? Again, any color there will be much appreciated.

And then maybe Gilmore for you on LCA-10 and IRD more broadly, can you just give us an update on what is a strategic option that you are exploring in the moment? What will be an ideal partner and ideal deal there? Thanks so much.

Baisong Mei

Thanks Luca, for your question. So just wanted to clarify. We have ambitious, but attainable goal to dose total of 20 patients by year-end. That is a separate from the registration of package. As Gilmore mentioned a bit earlier, that for the registrational package, a number of patients required and data package, we will need to actually have a engagement with the regulatory agency to finalize that.

Gilmore O’Neill

It is also worth adding that, the because we have actually agreement on the potency matrix or assay matrix that all the patients dosed in RUBY will actually provide data that will be part of marketing application.

With regard to your second question and strategic options for our IRD divestiture, we are actually exploring potential divestitures. An ideal partner would be a sponsor interested in rare ocular diseases and potentially with a particular interest in AV delivery.

But that is not a complete restriction, but an a key expertise in rare retinal disorders and their 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.

Luca Issi

Got you. Thanks so much.

Operator

Our next question is from Greg Harrison with Bank of America. Please proceed.

Greg Harrison

Hey, good morning. Thanks for taking the questions. First, just wanted to ask on expectations for the sickle cell data. I know it has been discussed previously, but just wanted to get your latest thoughts on, you know, 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 be complete with that?

Gilmore O’Neill

So with regard to the sickle cell data, again we were very encouraged by the initial clinical data, which were consistent with our preclinical data in demonstrating both impacts, robust effects on fetal hemoglobin and in the resolution of anemia. And we are looking forward and confident, we would see replication in subsequent patients for that data set and look forward to sharing updates in the middle and at the end of this year.

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 are looking for in that person leading forward. But the good news is that the discovery development, other functional components of the organization have good leadership and are already moving forward in the execution of our new strategy.

Greg Harrison

Great, that is helpful. thanks for taking the questions.

Operator

Our next question is Yanan Zhu with Wells Fargo. Please proceed.

Yanan Zhu

Hi, thanks for the questions. Have you dozed the third patient in the sickle cell trial yet? That is the first question. And at what point along the date of generation process from the sickle cell trial, do you plan to reach out and have a discussion with the regulator about the path forward for EDIT-301? And lastly, is the 301s manufacturing process carried out with a commercial grade process. And i.e., with some kind of bridging needed if it is not that kind of a process currently.

Gilmore O’Neill

Thanks very much Yanan. Baisong could you just sort of capitulate the update?

Baisong Mei

Yes. So thanks for your question, and I will get two first two questions. I will let Gilmore you answer the question about the manufacturer in there too. So, yes, and then we actually -- we did dose the third patient and started - as we mentioned that start the parallel dosing for the patients. And regarding the registration of path forward, we will discuss with health authority to finalize that data package required for registration, and we will share more data when the time is appropriate.

Gilmore O’Neill

Thanks very much Baisong. With regard to our 301 process, the good news is that the - certainly on the potency matrix area, we have actually been 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 agreed final commercial process that will be something that we will update you obviously at a time that is appropriate and obviously we can include more details.

Yanan Zhu

Great. Thanks for the color.

Operator

Our next question is from Gena Wang with Barclays. Please proceed.

Gena Wang

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 do you expect the transaction to be closed, does your cash guidance taking to consideration of impact from the deal? And that is a first question.

Gilmore O’Neill

Thanks, Gena. You are very good. You are going to keep them. That is 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 with the advance of the assets, the indeed that the - those - the impact in the financials is actually included or has been considered. So that is I think the first question.

Gena Wang

Okay. Have you already received the payment or you were received after the transaction close regarding the show?

Michelle Robertson

We have already - yes we have received the payment Gena.

Gilmore O’Neill

Yes. Actually, fair enough. I actually forgot to answer your part of your question, Gena, which is that we have closed the deal, we have received the payment. Yes.

Michelle Robertson

The payment was received in 2023.

Gena Wang

I see. So the cash reported already incorporate that.

Michelle Robertson

The cash reported at the end of the year did not yet include that.

Gena Wang

Okay. The guidance that will -.

Michelle Robertson

That will be in our reforecast of our cash runway.

Gena Wang

Okay. Sounds good. And the second question very quick, just I know you are committed to present a data 301 RUBY trial mid-year. What about the end of 2023, especially like ASH. Will you also provide some update there and thoughts on the timing and also number of patients?

Gilmore O’Neill

Yes. Thanks, Gena, I’m going to ask Baisong to answer that question, but confirm that we are planning to present updates both the middle of this year and at the end of this year. Baisong.

Baisong Mei

Yes, thanks Gena for the question. Yes, we will plan to have two data released for RUBY study. One is the middle of the year, and one is the end of the year. In terms of specific forum, we will share when we have more information.

And we have not shared the number patient this point, but I just mentioned earlier we really have a strong momentum for the RUBY study as well as EDITHAL study. And we expect to have multiple patient data for the data release.

Gena Wang

Okay. And then lastly, very quick, in-vivo programs beyond HSC, what would be your top organ to explore?

Gilmore O’Neill

Thanks very much, Gena. We actually have taken the approach that we will use a sort of a balance of multiple factors in selecting our targets, our therapeutic targets, and for ergo the target tissues. And that will be a balance of 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.

Gena Wang

Thank you.

Gilmore O’Neill

Thanks very much

Operator

Our next question is from Rich Law with Credit Suisse. Please proceed.

Richard Law

Good morning and thanks for taking my question. So as you focus on in-vivo development, is there any renew interest in using CRISPR/Cas9 in addition to Cas12 for internal product development? Since the Cas9 life technology is arguably more mature from the development progress by other companies. And then I have a follow-up question to that.

Gilmore O’Neill

Thanks very much, Rich. I have to say that our pipeline currently is focused on AsCas12a, we believe that the AsCas12a enzyme has some significant advantages. We actually also believe that now we have used it in the clinic. And actually, as Baisong has said, we have already edited multiple patients in addition to those already dosed. So we actually are building a clinical experience with AsCas12a.

And from the point of its advantages we believe that and actually have data supporting that position that it has a higher efficiency and essentially higher fidelity with much less off-target editing. So a substantial advantages for moving forward.

Richard Law

Right. Okay. A follow-up question to that is that, is there any performance requirement in the licensing agreement that you have with the broad, that you have to continuously perform product development with the Cas9 technology?

Gilmore O’Neill

I’m not going to go into details, but obviously, like any agreement there are conditions or requirements to execute. And ours are a mixture around both on our business development and execution.

Richard Law

Okay. Great. Thank you.

Operator

Our next question is from Madhu Kumar with Goldman Sachs. Please proceed.

Unidentified Analyst

Hey, thanks for taking our question. This is [Rob] (Ph) on for Madhu, two quick ones. What venue should we expect the mid 2023, 301 update, it is like medical meeting or company release. And then how do you think about the regulatory path in in-vivo CRISPR in the U.S. given the headwinds we have seen by some of your peers? Thank you.

Gilmore O’Neill

Baisong, do you want to take the first question? I will take the second.

Baisong Mei

Sure. Yes. So we plan to release the RUBY data middle of the year, as well as end of the year. The specific venue we have not decided, we will share in the later time.

Gilmore O’Neill

Thanks very much, Baisong. 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 will say that one of the things that drew me to Editas was the substantial investment and depth of expertise built in both its research analytics, as well as the CMC analytics from the point of view of certainly managing risk, et cetera.

And so I actually feel, you know, very good about where we are, from that point of view. And obviously as we move forward, we will engage with regulators, including the FDA to determine the path to our first-in-human.

Unidentified Analyst

Thank you.

Gilmore O’Neill

Thanks very much.

Operator

Our next question is from Matthew Harrison with Morgan Stanley. Please proceed.

Unidentified Analyst

Thanks for taking our questions. This is [Chris] (Ph) 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 is 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.

Gilmore O’Neill

Thanks very much. I think - let me take both questions regarding the filing 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 is only something that we will know as we progress in negotiations with them.

With regard to the BMS collaboration we are happy with the collaboration. BMS 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. As I say, we are very excited by the collaboration and delighted to see the progress.

Unidentified Analyst

Thank you very much.

Operator

Our final question is from Liisa Bayko with Evercore ISI. Please proceed.

Liisa Bayko

Hi, all my questions has been answered, but thanks for taking me in.

Gilmore O’Neill

Thanks Liisa

Operator

Thank you. This will conclude today’s conference. You may disconnect your lines at this time and thank you for your participation.