Q1 2024 Editas Medicine Inc Earnings Call

Participants

Christy Barnhart; IR; Editas Medicine, Inc.

Gilmore O’Neill; CEO, President; Editas Medicine, Inc.

Baisong Mei; EVP, Chief Medical Officer; Editas Medicine, Inc.

Linda Burkly; EVP, Chief Scientific Officer; Editas Medicine, Inc.

Erick Lucera; EVP, CFO; Editas Medicine, Inc.

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

Samantha Semenkow; Analyst; Citigroup Inc.

Joon Lee; Analyst; Truist Securities, Inc.

Mary Kate Davis; Analyst; Bank of America

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

Gena Wang; Analyst; Barclays Bank PLC

Mani Foroohar; Analyst; Leerink Partners LLC

Brian Chang; Analyst; JPMorgan Chase & Co

Eric Schmidt; Analyst; Cantor Fitzgerald

Jay Olson; Analyst; Oppenheimer & Co. Inc.

Yanan Zhu; Analyst; Wells Fargo Securities, LLC

Luca Issi; Analyst; RBC Capital Markets

Presentation

Operator

Good morning and welcome to the Editas Medicine first quarter 2024 conference call. (Operator Instructions) Please be advised that this call is being recorded at the company's request. I would now like to turn the call over to Christy Barnhart, Corporate Communications and Investor Relations at Editas Medicine.

Christy Barnhart

And thank you. Good morning, everyone, and welcome to our first quarter 2024 conference call. Earlier this morning, we issued a press release providing our financial results and recent corporate updates. A replay of today's call will be available in the Investors section of our website approximately two hours after completion. After our prepared remarks, we will open the call for Q&A.
As a reminder, various remarks that we make during this call about the Company's future expectations, plans and prospects constitute forward-looking statements for the purposes of the Safe Harbor provisions under the Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including those discussed in the Risk Factors section of our most recent annual report on Form 10-K, which is on file with the SEC as updated by our subsequent filings.
In addition, any forward-looking statements represent our views only as of today and should not be relied upon as representing our views as of any subsequent date, except as required by law. We specifically disclaim any obligation to update or revise any forward-looking statements even if our views change.
Now I will turn the call over to our CEO, Gilmore O’Neill.

Gilmore O’Neill

Thanks, Christy, and good morning, everyone. Thank you for joining us today. On Editas is first quarter 2024 earnings call. With me today are four members of the Editas executive team our Chief Medical Officer, Baisong Mei; our Chief Scientific Officer, Linda Burkly; our Chief Financial Officer, Erick Lucera; and our Chief Commercial and Strategy Officer, Karen Deardof.
We are pleased with Editas has momentum and progress in the first quarter of 2024. It has the stories to deliver life-changing medicines to patients with previously untreatable or undertreated genetically determined diseases and our vision and focus strategy is to position a test as a leader in in vivo program with gene edits.
Three Pillars underpin our strategy. The first of those pillars is to drive ready cell and edited cell therapy for hemoglobinopathies and formerly known as editorial one toward BLA and commercialization. Second is to build an in vivo editing pipeline. And the third is to increase business development activities with a particular focus on monetizing our very strong intellectual property.
At the start of 2024, we announced the following 2024 objectives for Randy. So we will provide a clinical update from the Ruby trial for severe sickle cell disease and the endometrial trial for transfusion dependent beta-thalassemia in mid-2024. And by year end 2024, we will complete adult cohort enrollment and initiate the adolescent cohort in Ruby and continue enrollment in editor for our in vivo pipeline, we will establish in-vivo preclinical proof of concept for an undisclosed indication. And for BD, we will leverage our robust IP portfolio and business developed to drive value and complement core gene editing technology capabilities. So how we executed against this strategy and these objectives in the first quarter.
Let us start with Renaissance first on enrollment. We've been very pleased with the growing patient and healthcare provider interest in Renaissance. Indeed, we are delighted to share that we have completed enrollment in the adult cohort of the Ruby clinical trial. Additionally, we have enrolled multiple patients and have more in screening in the adolescent cohort of a Ruby study, which was launched at the beginning of this year. And we continue to enroll beta-thalassemia patients in our adult study. Dosing continues in both the Ruby and NFL studies.
Second, on clinical data, we remain on track to present a substantial clinical dataset of at least 18 sickle cell patients with 2 to 21 months of clinical follow-up in the Ruby study in the middle of 2024, and we will share a further update by year end. We are also on track to present clinical data from the at-home study of red cell transfusion dependent beta-thalassemia in the middle of 2024 and again by year end, based on my will share more ready foundations later on in this call.
On the manufacturing front, I am pleased to share that we have promoted Greg Whitehead to the role of Chief Technology and Quality Officer, leading our technical development, technical operations and quality departments. Greg has more than 25 years of experience in the biotech industry has extensive cell and gene therapy, clinical and commercial development expertise.
Now let's turn to in-vivo and our pipeline development, where we continue to strengthen our in vivo discovery capabilities and continued lead discovery work on in vivo therapeutic targets in hematopoietic stem cells and other tissues. Importantly, we remain on track to establish in-vivo preclinical proof-of-concept for an undisclosed indication by the end of the year. Our internal development efforts are differentiated by leveraging the Indo crisper technology.
We already used to up-regulate gamma globin expression through direct debiting of the HBG. one two promoter size in our ex-vivo ready cell program. Our in vivo approach is aimed at functional up-regulation of gene expression in genetically defined diseases with a preliminary focus on rare and orphan patient populations in the medium to long term, we intend to expand to more common genetically determined diseases. Linda Berkeley, our CSO, will share more details on our in vivo strategy and progress towards building an in vivo pipeline later on in the call.
Finally, what is happening in business development. In March, we signed a two year extension to the collaboration with Bristol-Myers Squibb to research, develop and commercialize autologous and allogeneic alpha-beta T cell medicines for the treatment of cancer and autoimmune diseases. We also have options to extend that collaboration for an additional two years to date, Bristol-Myers Squibb has opted into 13 different programs across 11 gene targets to date, two programs are currently in IND-enabling studies and four programs are in late-stage discovery and the intellectual property.
Yesterday, oral arguments were held before the US Court of Appeals for the Federal Circuit regarding an appeal of the Patent Trial and Appeal Board or PTAB, previous decision favoring Broad Institute in the US patent interference involving specific patents for crisper Cas nine editing in human cells between the University of California, University of Vienna and Emmanuelle sharp on TA for CDC. and the broad, we expect a decision on the case in the second half of 2024.
Eric will share more BD. and IP. details later on in the call. We are energized by our progress and execution this quarter with our sharpened strategic focus, our world-class scientists and employees, our team drive and execution and strong balance sheet. We continue to build momentum to progress our strategy to deliver differentiated medicines to patients with serious diseases.
Now I will turn the call over to Baisong, our Chief Medical Officer.

Baisong Mei

Thank you, Guillermo, and good morning, everyone. Let's talk about Renaissance, which is under clinical development for severe sickle cell disease and transfusion dependent beta-thalassemia. As Guillermo shared, we are pleased that we have completed enrollment in the adult cohort of the Phase one two three will be trial and the dosing continues in the adolescent cohort of there will be a study where we enrolled multiple patients and several more patients in screening, the interest and demand are high and very pleased about how quickly we have moved in screening and enrollment of the adolescent cohort.
I'd like to thank colleagues and editors and our clinical trial partners for the collaboration and hard work. And more importantly, I would like to thank the patients, their families, investigators and study site staff for their trust and support in the ADHERE trial for transfusion dependent beta-thalassemia. We continue to move forward with enrollment and dosing. We look forward to sharing clinical data in the mid of this year and also at the year end, as I have shared.
I visited and continue to build our Ruby in additional clinical trial sites and speak with the investigators. I appreciate the enthusiasm and support from investigators and study size. I'm pleased with the momentum of Renevia in patient recruitment freezes and dosing in both studies. I'm excited to hear from the investigators that patient goes to the rental sale have already seen positive changes in their lives.
As we shared in our February earnings call, we aligned with FDA that will be clinical trial is now considered a Phase one two three trial for BLA filing. We also have alignment with the FDA on the study design endpoints and sample size. We look forward to future discussions with FDA and continue the calibration.
Turning to clinical data, as Gil mentioned, we are on track to present a substantive clinical data set of sickle cell patients with considerable clinical follow-up will be study in the middle of 2024 and a further update by year end 2024. What do we want to show there will be data set, including clinical data from at least 18 sickle cell patients with a 2 to 21 months of follow-up. And the editorial dataset waiting for the clinical data from seven patients with a four to months follow-up, we will present efficacy data, including total hemoglobin, fetal hemoglobin and the vessel completed events or VOE.
For sickle cell patient in will be studied and red blood cell transfusion for transfusion dependent thalassemia patients with PTCL study and safety data, including mutual appeal and probably engraftment for both steady. As a reminder, in December 2023, we shared safety and efficacy data from 11, we'll be patient and six additional patients. Once again, the data confirm the observation from our prior clinical readouts, including sales drilled only reflect the correction of anemia to a normal physiological range of total hemoglobin in sickle cell patients went out to a robust and sustained increase in fetal hemoglobin level in excess of 40%.
All Ruby sickle cell patients remained free of battle group events following Renaissance treatments. When you say are treated as because the location and transfusion dependent beta-thalassemia patients have shown successful engraftment have stopped the red blood cell transfusion and the safety profile of Renault sales to date is consistent with both of them, ablative conditioning and the autologous hematopoietic stem cell transplant.
These data reinforce our belief that we have a competitive product and a product potentially differentiated from other treatments with a rapid correction of needs, thanks to the deliberate choice of our discoveries have made early in the program, the choice of critical enzyme and the targeted two edits for increased fetal hemoglobin expression matters. Retail uses our proprietary, a scaffold A. enzyme to upregulate the HB. two and two promoter. Based on the clinical data thus far, we believe that sustained normal levels of total hemoglobin could be a potential point of differentiation for Renevia.
Now I turn the call over to Linda, our Chief Scientific Officer space on Emblem.

Linda Burkly

Good morning, everyone. I'm happy to be talking to you this morning to share more details about our in-vivo strategy and our progress towards building an in vivo pipeline. I would like to take a moment to remind you why we believe in vivo medicines will be a disruptive transformative development in medical history with the potential to address genetically determined diseases with durable and curative outcomes for patients. First, in vivo medicines may reduce the administration burden of delivering editing medicines to patients in need, which will provide a broader access to patients all around the world.
Second, off-the-shelf administration may allow for scalable manufacturing and lower cost to produce based on these two principles. We believe that in vivo gene editing will provide accessible cures for genetic diseases and therefore, it may be the most disruptive development in medical history.
So how will Editas position itself. There are many monogenic diseases that can potentially be cured with the gene editing approach. We have said that we will first target the development of treatments that are clearly differentiated from current standard of care, and that will leverage the aspects of crisper editing that give it a unique advantage over other therapeutic modalities.
Our internal development efforts are differentiated by leveraging Intel crisper technology reasons to up-regulate gammaCore globin expression through direct editing of the HBG. one two promoter site in our XC low renaissance program. Our in vivo approach is aimed at functional upregulation of gene expression in genetic diseases in rare and orphan patient populations from which we intend to expand to more common diseases. I'm also pleased to share several progress updates as we advance our in-vivo capabilities towards our long-term vision of being a leader in in vivo program, both gene editing.
First and most importantly, as Gil mentioned, we remain on track to establish in-vivo preclinical proof of concept for an undisclosed indication by the end of the year. Editas is well positioned with established capabilities in the four main components of in vivo gene editing medicine, one guide RNA, two editing enzymes, three messenger RNA and four delivery technologies. And we are currently evaluating lipid nanoparticles for delivery of gene editing cargo into multiple tissue types with multiple companies. Additionally, we are evaluating next-generation delivery technology.
Second, our in-vivo capabilities with the potential to be used in developing transformative in vivo gene editing medicines are demonstrated by the preclinical data we are presenting at the American Society of Gene and Cell Therapy or ASGCT Annual Meeting in three presentations taking place on Thursday and Friday of this week on Friday, in an oral presentation, we will share in vivo preclinical data from mouse studies using lipid nanoparticle mediated delivery of an optimized guide RNA and engineered AS. casualties messenger RNA in poster presentations on Thursday and Friday, we will share preclinical data demonstrating a F. capital, a guide RNA modifications.
It enables high potency gene editing in multiple cell types, including in the liver and improve gene editing outcomes in vivo, enabling the development of in vivo gene editing medicine and research on identifying potent large hearing companies is LSR as a foundation to develop novel in vivo gene editing technologies for holding knock-in, expanding potential in vivo gene editing targets for developed medicines. Third, edit test crisper based in vivo gene editing capability has been clinically validated. Notably in 2020, I had us as the first company ever to treat human with an in vivo delivered crisper based gene editing medicine at it one on one.
In fact, earlier this week, the New England Journal of Medicine published a manuscript entitled gene editing, Percepta 90 associated retinal degeneration, detailing our formally development candidate that it went to one for the treatment of Leber's Congenital Amaurosis type 10 or LCA10. It is established clear in vivo human proof of concept in 2022.
And we are pleased that the results from this groundbreaking clinical trial were published by the New England Journal of Medicine. These progress updates demonstrate Editas execution on our in vivo strategy and our proven in vivo gene editing capabilities. And I look forward to sharing more details about our in-vivo development strategy and our progress towards building an in vivo pipeline later this year.
Now I will turn the call over to Eric, our Chief Financial Officer.

Erick Lucera

Thank you, Linda, and good morning, everyone. I'm happy to be speaking to you, and I'm excited to provide updates on our business development achievements, intellectual property and financial results for the first quarter of 2024. First, in regard to business development, as Gil mentioned, in March, we announced a two year extension to the collaboration with Bristol-Myers Squibb to research, develop and commercialize autologous and allogeneic alpha-beta T cell medicines for the treatment of cancer and autoimmune diseases. We also have options to extend the collaboration for an additional two years to date, Bristol-Myers Squibb has opted into 13 different programs across 11 gene targets.
To date. New programs are currently in IND-enabling studies and four programs are in late stage discovery. As a reminder, for each new experimental medicine, the Bristol Myers Squibb develops and commercializes using opted into genome editing tools, investors, Bristol-Myers Squibb will pay Editas Medicine and potential future milestone payments following the approval of any products resulting from the collaboration. Editas Medicine is also eligible to receive tiered royalties on net sales.
So we are pleased that our Bristol-Myers collaboration has proved to be a productive partnership, and we are committed to future collaborations and partnerships that will allow for the continued access and advancement of gene editing and in IP. As Joe mentioned yesterday, the oral arguments were held before the US Court of Appeals for the Federal Circuit.
Regarding the CBC.'s appeal of the PTAB.'s decision involving patents for crisper cash, nine editing in human cells. As you know, the Broad Institute has previously prevailed three times against the CBC twice with the PTAB. And once at the Federal Circuit, the Federal Circuit's review will determine whether the PTAB. correctly applied the law. It is important to remember, the court will not hear new evidence, an appellate court decision in the broad favor would reaffirm Editas position as the exclusive licensor of the patents covering [cash nine] used in human medicines in the US.
Is also important to remember that only a small fraction of the IP we licensed from the road are involved in the ongoing USPTO interference proceedings. We expect a decision on the case in the second half of 2024, we remain confident that the growth will once again prevail our IP portfolio of foundational US and international patents covering cash, nine and cast 12 used in human medicines are a source of meaningful value as we believe that globally there are more than 100 cash noncash 12 A.
Programs in development worldwide with the majority of the programs being developed by 10 companies. We believe these potential fields represent a potential material source of non-dilutive capital as evidenced by our deal in the fourth quarter of 2023 that extended our cash runway by two quarters. We look forward to future discussions and now 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 2024.
I'll take this opportunity to briefly review a few items for the quarter. Our cash cash equivalents and marketable securities as of March 31, $377 million compared to $427 million as of December 31st, 2023 we expect our existing cash, cash equivalents and marketable securities, together with near term annual license fees and contingent upfront payments payable under our license agreement with Vertex to fund our operating expenses and capital expenditures into 2026.
Revenue for the first quarter of 2024 was $1.1 million compared to $9.9 million for the same period in 2023. A decrease relates to the January 2023 one-time sale of the Company's wholly owned oncology assets and related licenses. R&D expenses this quarter increased by $11 million to $49 million from the first quarter of 2023.
This increase relates to additional clinical and manufacturing costs that support the continued progression of the Company's red cell program. The increase is also attributable to one-time payments related to sublicense and license obligations and costs will continue to incur these types of payments as we and our collaboration partners advanced certain license programs in the gene editing space.
G&A expenses for the first quarter of 2024 were $19 million, which decreased from $23 million in the first quarter of '23. The decrease in expenses primarily attributable to one-time professional service expenses related to the 2023 strategic initiatives and business development activities, as well as reduced legal and patent costs with our BD. and IT. activity and a cash runway into 2026.
Editas remains in a strong financial position. We have ample resources to continue the advancement of our Renaissance program, support the progression of our in vivo capabilities to develop our pipeline and leverage our strong IP position for additional business development and licensing opportunities. For that, I'll hand the call back to Gilmore.

Gilmore O’Neill

Thank you, Eric. We are proud of our progress in the first quarter of 2024. I look forward to continue to accelerate the momentum in 2024 as we continue to evolve from a development stage technology platform company into a commercial stage gene editing company. We look forward to continuing our transformation and sharing our progress with you. As a reminder of our 2024 strategic objectives for any sale, we will provide a clinical update from the Renaissance Ruby trial for severe sickle cell disease and the NHL trial for transfusion dependent beta-thalassemia in mid-2024 and year end 2024.
We have now completed the adult cohort enrollment and have started enrolling patients in the adolescent cohort and Ruby. We will also continue enrollment in FL and dosing in both trials. For our in vivo pipeline, we will establish in vivo preclinical proof of concept for an undisclosed indication. And for BG., we will leverage our robust IP portfolio and business development capabilities to drive value and complement core gene editing technology capabilities.
As we shared today, we are making significant progress in all three pillars of our strategy this quarter, including red cell in vivo and business development, including intellectual property. We entered 2024 with great momentum, and I am proud of the Editas team significant progress towards becoming a commercial-stage company and on developing clinically differentiated transformational medicines for people living with serious previously untreatable diseases. As always, we could not achieve our objectives without support of our patients, caregivers, investigators, employees, corporate partners, and you.
Thanks very much for your interest and attention, and we're happy to answer questions.

Question and Answer Session

Operator

(Operator Instructions) Samantha Semenkow, Citi.

Samantha Semenkow

Hi, good morning and thanks very much for taking my questions. And I'm wondering about your in vivo pipeline and the proof of concept that you're expecting by the end of this year, a what will be the bar for success that you're looking for in this program?

Gilmore O’Neill

Thank you.
Thanks very much, Samantha. I'm going to ask Linda to address that.

Linda Burkly

Thank you, Sam. Thank you for the question. We are looking for a proof of concept for high efficiency delivery and editing for our target of interest in vivo in this preclinical POC that will give us confidence in our ability to target the target of interest. We are going to be sharing more information on this at a future date.
Thank you for the question.

Operator

Joon Lee, Truist Securities.

Joon Lee

And congrats on the progress and thanks for taking our question. An interesting update on your disclosure. Is there a plan to identify large ceiling combinations, which implies sort of a newer approach to many of you that many of the peers are also developing some call it team adding some call it pasting does the size of the Company's media and insertion allow for you starting a coding sequence for dystrophin, for example, and are you able to comment on whether TMD is out of the question regarding your in vivo aspirations? Thank you.

Gilmore O’Neill

Thanks very much, June. I'm going to have Linda sector.

Linda Burkly

Yes, thank you for commenting. We're excited about the LSRI. technology that we're disclosing here. We are identifying many different novel LSR. We're not disclosing at the moment. The size of the integrations that can be accommodated by these large Jeremy companies is, but we have quite a few novel LSR.s that we've identified and we're further characterizing them. And thank you for the question.

Operator

Mary Kate Davis, Bank of America.

Mary Kate Davis

Good morning. Thanks for taking my question. I'm looking at the red cell program here. How are you guys looking at the midyear? Any follow-up data compared to the year-end update here as far as time progresses, what should we look for from treated patients in terms of safety and efficacy moving forward.

Gilmore O’Neill

Thank you.
Thanks, Mary Kay song is going to take that one.

Baisong Mei

Thanks, Mary. And so we're in the middle year, our release we expect to have at least 18 patient data for Ruby study and within the 18 patients, and 4 of them will have 12 or longer 12 to 21 months of exposure and seven were out 5 months to 12 months exposure, another 7 with 2 to 5 months exposure. With that, we are we feel this data is very meaningful to see that direction, not only the increase of total hemoglobin normalization of our top total hemoglobin increase of fetal globin.
But also the durability of the study and impact our from an efficacy perspective for, for example, the free of big vessel currency events for the Adacel study, we'll have patients perform at least seven. Those patients. We have four to 12 months of exposure, which also where we have a meaningful variance compared to the December release, we have 11 will be patient and six editorial patients. So their substantial more data will help us to understand the program much better.

Operator

Jack Allen, Baird.

Jack Allen

And congratulations to the team on the progress, and thanks for taking the question. I'm going to stick with the red cell program. I was hoping you could provide some color on dosing of the pivotal cohort. I know you've commented on the adult cohort being fully enrolled but have all those patients receive therapy.
And any other comments you can provide as it relates to the size of the cohort that you've agreed to with regulators would be very helpful. Thanks so much.

Gilmore O’Neill

Thanks very much, Jack. I'll answer the first part and then pass it to Basil to back, you know, maybe give us some more color to regulatory interactions we have obviously completed the adult enrollment, which are actually very excited about not least because that is in the context of two approved therapy. So it really is a very concrete reflection of the enthusiasm that they saw has found and described, indeed, increasing enthusiasm about our program with his visits to sites and conversations with investigators.
Our health care providers and indeed with patient advocacy groups, Tom, we have scheduled that many of those patients already for dosing, and we'll give you further updates on the progress of dosing at a later date. And with regard to the regulatory color, I think Basil, you might want to maybe just share a little more of that.

Baisong Mei

Yes, for regulatory, as we shared that, we have alignment with FDA that there will be study is a Phase one two three study for PMA filing. And we also have alignment on the sample size, duration and study design of that. So we continue to have collaboration with FDA on the further discussion about this progress.

Jack Allen

Great. Thank you.

Operator

Gena Wang, Barclays.

Gena Wang

Thank you. If I may very quick two questions. First, should we read into your ASGCT. presentation for in-vivo indication such as glaucoma? And second, I know you mentioned also a little bit. But when we look at the current approved genomic therapy for sickle cell, we still have a 10% patient relapse with VOD events. What do you think is the key factors that we should look into for the potential of differentiated durability was hopefully 100% control.

Gilmore O’Neill

Thanks very much.
Gena. I'm going to ask Linda to handle the first part of your question around the ASGCT. and then based can talk about durability, what we're seeing to date have been.

Linda Burkly

Thank you very much, Gena, on primary open-angle glaucoma is obviously an area of very high unmet need. And while we conducted those studies because of that, we are not currently pursuing that indication, but we were able through those studies to really demonstrate impressive preclinical POC and showcase our in vivo capabilities with respect to three components are our ability to deliver lipid nanoparticles in vivo with our gene editing cargo, our proven capability of our proprietary enzyme and Kestrel way to edit in vivo and our guide modifications to enhance and gene editing potency. So these capabilities really position us well for delivering in vivo gene editing medicines, and we're thrilled pleased with the ASGCT. disclosures.
Thank you.

Baisong Mei

Yes, I can. I will tick up tick up on the question about the BOE. relapse for sickle cell patients. And first, I would like to say I want to congratulate the entire field of effort, the treating sickle cell disease including the recent approval of the two molecules. And so I think what we see is that with the continued effort of all of us, we will continue to improve and transform the treatment for sickle cell disease patients with renal cell.
And we are still continuing to collect the clinical data. As I mentioned, we expected this is a different not only competitive but differentiated molecule with the normalization of the total hemoglobin correction of pneumonia. So we're looking forward to see our own data on the VOE. as we reported so far we have seen all those patients, those with Renault sale, the three BOEB.

Gena Wang

Thank you.

Operator

Mani Foroohar, Leerink Partners.

Mani Foroohar

Hi, good morning. This is CJ on for money and thanks for taking your question on ours is following the agreement with Vertex. Can you just how you're thinking about future IP monetization opportunity.

Gilmore O’Neill

Thanks very much. I'm going to ask Eric to address that question.

Erick Lucera

Yes, thanks for the question. Obviously, as we said in the transcript, we view the future potential royalty monetization and licensing activities as an integral and very important source of non-dilutive capital. As you know, these are foundational IP patents which are applied to just about everybody's projects in cash, non-cash 12 and we expect to have conversations with those folks as soon as we can.

Mani Foroohar

Thank you.

Operator

Brian Chang from JP Morgan.

Brian Chang

Hey, guys, thanks for taking our question this morning. Can you just remind us what's your latest thinking around the timing of holding discussion with regulators on sickle cell? And, you know, just given the data that you're going to present mid year. Any update and color on the timing from holding a productive conversation with regulators would be appreciated. Thank you.

Gilmore O’Neill

Thanks very much, Brian. Going to us based on to talk about that sort of. I think your two questions really, which is about what the data are in the middle of the year and how they integrate with our discussions with regulators.

Baisong Mei

So Brian, we as I mentioned, we are very pleased with the data we're going to release the middle of the year and which is substantive and also give us good direction how much you will we will get and to have, for example, a dataset to have equivalent to the SGVIBOF. filing, for example. So that's kind of one part of that where they happy to see the amount of data and the patient outcome on the data.
And then the other thing is by the regulatory engagement. As I mentioned, we already have a line of this is Phase three study to support the BLA. And we have then we have our continued engagement with the with FDA. We have not disclosed all those details of the interaction yet.
But as a reminder, we have our MET explanation and which allow us to have frequent interaction with agency, but also with a high level, our interaction with the agency. And this, of course, helps give us opportunity for potential priority review and rolling submission. So we have been excited to the direction we continue to have engagement and cooperation with FDA.

Operator

Eric Schmidt, Cantor Fitzgerald.

Eric Schmidt

Thanks for the question. And congrats on the progress on. Are you able to give the approximate number of patients who were enrolled in the Ruby trial with sickle cell disease? And then it sounds like you've been able to make pretty good progress in enrollment in that study despite the availability of commercial cell therapies. Just wondering if at centers that have both experimental and commercial cell therapy available may be based on could talk a little bit about what's driving the decision to use the Editas product over others. Thanks it.

Gilmore O’Neill

Thanks very much, Eric. And based on we can update you on where each of our clinical trials, trials.gov set as a target for the cohort in our trial and then obviously build on his perceptions of why we're doing so well with enrollment even in the context of commercial therapies being available.

Baisong Mei

Thanks, Kyle. We are very pleased with the momentum by the enrollment in both the adult cohort and the adolescent cohort. And for the adult cohort, we and we shared that in February, we dosed We enrolled 40 patients now in or slightly more than 40 patients and therefore, we close the the adult cohort you employment. And for adolescent cohort, we started like beginning of this year, we already enrolled a modification and have modification and screenings were very pleased with that.
Then, as I mentioned, I'm on the road all the time and to visit our investigators and the study size. And then they really feel that one is actually there, I believe on the Renault sale based on the MOA based on the data, we have continued to sharing on that. I also do credit the entire field working on that with a two gene therapy approved for sickle cell is also increased the interest in the direction of the gene therapy for sickle cell disease. So that's how we see you know, over the last year or so, we see really great momentum that for Renaissance enrollment, especially after we release our data.

Operator

Jay Olson Oppenheimer & Company,

Jay Olson

Congrats on all the progress, and thank you for taking the question. Can you talk about the timing of the collaboration extension with Bristol? Was there some new data that that triggered the new collaboration? And is there any color on what new data Bristow may have seen? And then And separately, can you talk about any work that you've done on developing a milder conditioning agent.

Gilmore O’Neill

And what I want to do is ask Eric to address the question about the BM. What I do want to say I can address the conditioning, which is that just at our last earnings call, we talked that we are going to continue monitoring the space. We have significant contacts in the academic and nonacademic world around the field, but we have actually really deployed our efforts and our resources internally to focusing on our in vivo pipeline, including developing hematopoietic stem cells.
The rationale for that being that we see that where a milder conditioning therapy is actually approved, it would be used universally adopted universally in transplant centers across multiple indications, including on stem cell transplantation. Hemoglobinopathies. And with that software might I'm just going to pass to Eric just to talk about the BMS I deal.

Erick Lucera

Yes, thanks for the question. With respect to the timing of the renegotiation or the extension. Obviously, we put out a press release in the very recent past a week or two ago, something like that. That would give you an update on the timing, I would say with respect to the data, Bristol Myers, as you know, recently completed a portfolio review and we were pleased to see that all of the projects that we're working on them are continuing to move forward. I think we would leave discussion of specific programs to them to talk about a thing that they're seeing in those programs.
I would highlight the fact that at their most recent R&D Day last September, which I think was the first one they've done in several years, they did mention six products on their pipeline chart which are using our technology. So I would refer you to their R & D disclosures from that meeting to get an update on the work that they're doing with us, but we are very excited about working with them. This has been a partnership that has survived several mergers and several portfolio reviews. So we're very excited about what we're seeing Thank you.

Operator

Phil Nadeau, TV Cowen.

This is Alex on for Phil. Thanks for taking my question. So given the association between total hemoglobin hemoglobin levels and organ function. Do you plan to utilize any quantitative endpoints on basically assessing and organ function in the REVEAL trial? And if so, what might those look like? And when could we maybe expect initial data? Thanks.

Gilmore O’Neill

Thanks very much, Alex. I'm going to ask based on to address that question.

Baisong Mei

Yes, thanks, Alex. We certainly have measurements for the end organ function are we looking to the several major organ system to monitor the function improvement? For example, we monitor the liver function, not only with these different lab values. We're looking to our pulmonary function to check to check the the respiratory system.
And we are we also have cardio ankle and other measures. You're talking to measure their cardiovascular system and that too. So we are looking forward to see more data on that and give us more understanding of the end organ. It's a function may be may behave after the treatment Just a reminder that we also, of course, look into that not only sickle cell but also other area in this in terms of the anemia, how that impact a function and how that correcting anemia may be able to improve that assumption after the after treatment in sickle cell.
Specifically over the last couple of years, you already see more publications about end organ function even after the allogenic transplant to treating sickle cell patients. We are very excited on that. But just to be very honest to ourselves, right, this field is still fairly new and we see some really good publication and direction in this, and we're looking forward to our study as well as the literature on this field.

Okay, thank you.

Operator

Yanan Zhu, Wells Fargo.

Yanan Zhu

And thanks for taking our questions. So first, on the differentiation of total hemoglobin normalization, I was wondering, have you had feedback from sickle cell treaters on that differentiation and whether there's any hesitancy or pushback that perhaps the current level of hemoglobin achieved by the marketed product is sufficient and what how much of that kind of thinking is it out there? And on the in vivo side, was wondering are you focused on first-in-class targets or perhaps I not first-in-class targets, but hoping to have a differentiation on specificity and transduction efficiency, et cetera.

Gilmore O’Neill

Thanks very much. And I'm so I'm going to ask a song to talk about the differentiation of total hemoglobin. And then I'll ask Linda, just to talk about our approach to first in class or clear differentiation and clear that we would see that with our approach to function of regulation. Thanks.

Baisong Mei

Thanks, Alan. I mean, certainly we talk about the investigators as well, the KOLs and sickle cell treaters for differentiation and how we may be able to see and with the position of this molecule. And when we're talking to a loosely hematologist and sickle cell treaters, they see our data. And when once the hematologist mentioned that it is a no-brainer that it's better if you have a 16 gram, but that's the leader versus 10 gram, but definitely the of total hemoglobin.
And then they also I mentioned the enthusiasm of our study and they have finally been knowledgeable hematologist. They see the difference among several molecules. And we investigator said that he was waiting for our trial and we did not anticipate other. So those other anecdotal examples on that. As I mentioned earlier, we certainly want to look forward to see the clinical data.

Gilmore O’Neill

It's also worth highlighting, of course, that the FDA has recognized that a one gram per deciliter difference is meaningful or certainly likely to predict a clinically meaningful benefit in it. They used that threshold to be of an expenditure approvals to Oxbryta in the past Yes.

Baisong Mei

And talking about that at least is also when we communicate with FDA, this is also appointed. We have been discussion with FDA.

Gilmore O’Neill

So thanks very much based on, Linda, just VRD in vivo and where our focus is.

Linda Burkly

Yes, in vivo, our in vivo approaches aimed at functional upregulation of gene expression, genetically determined diseases. And this strategy positions us very well to be differentiated from others in terms of our targets and our target editing strategies.
And what this means is that we can go after targets that others can't go after and so from an indication perspective, we can go after indications that perhaps others can go after. And so we could have a first-in-class strategy also within a given indication, we could devise a targeted strategy that would be it's best in class, if you will. So we can have first-in-class strategies as well as best in class opportunities. I hope that answers here.

Yanan Zhu

Yes, thank you. Very helpful. Thanks for all the answers.

Operator

Luca Issi, RBC Capital.

Luca Issi

And congrats on the progress. And maybe just a quick one on red cell and the filing strategy. What's the vision here for DNA? Are you planning to file adults and adolescents concurrently or sequentially. Any color there?
Much appreciated. And then maybe quickly on the Middle East, can you just talk about the opportunity for sickle cell disease in the Middle East on Vertex? Seems really, really excited about that market. So wondering what's your strategy there to potentially tap that market? Are you still focused on partnership? Can you potentially access that via distributor again, any thoughts there much appreciated. Happy to department.

Gilmore O’Neill

Thanks very much, Luca. So I'll ask based on just talk about our regulatory strategy as far as we have shared it. And then Karen can talk about just how we're looking at and thinking about the Middle East and frankly, in the context of the rest of world.

Baisong Mei

Yes. Yes. Thanks, Luka up, we are very pleased with the interaction with the agency and continued interaction with the agency about the rental sale. New for the there will be study as a Phase three study to support the BLA and of all the front of the Phase three study.
We have alignment of that and we have continuously a conversation with FDA on this route. We have not shared the specifics about the date of the BLA or the indication of it out of a alone or adolescent. But as we shared, we are very pleased with the enrollment for close our cohort as well as adolescent cohort. So that give us a great position for this market.

Luca Issi

Thanks.

Linda Burkly

Great, Luca, thanks for the question about Middle East and certainly just the populations outside the United States. And there's absolutely a number of geographies where there is a significant population of sickle cell patients and really high unmet need of beta-thalassemia as well.
What I'd say is our continued drive to execute in the U.S. and to move ourselves forward with differentiation just continues to support the opportunity for us to partner at the appropriate time. And that's certainly something that we've said we can we are open to, and we'll certainly provide more more color in the future as appropriate.

Luca Issi

Thanks so much.

Operator

Steve Seedhouse, Raymond James.

Thank you. This is Nick on for Steve. We actually have a longer-term question. To what extent can you leverage the infrastructure that Vertex is already building out for cash given will will added through I won't be able to plug into the existing authorized treatment centers once launched. Thank you.

Gilmore O’Neill

Thanks very much and thanks very much. And Nick, I'm going to ask Karen to address that.

Caren Deardorf

Thank you, Nick, for the question. In a first, we'd say I'd say that I'm really pleased to see some of the initial progress for the other therapies and being able to get patients started. And we always anticipated that it would be a debate, many centers starting to dip their toes on as they build the infrastructure and they gain the confidence. So to answer your question, absolutely, we've always said that in this kind of market of the complexity of the ex vivo being a fast follower is absolutely an advantage.
And we we also, on our own have a really strong base of over 20 clinical sites in the U.S. was very strong enrollment and relationships that we're leveraging and those relationships and the guidance they're giving us will be really pivotal for us as well. But this is a field that will benefit from the increase in education, which patients which based on mentioned also helps with our enrollment and just building the infrastructure. But we are engaged on the KOL, the patient advocacy as well as on the payer front on to ensure that we're prepared. Thanks for the questions.

Thank you.

Operator

Jack Allen, Baird.

Jack Allen

Great. Thanks so much for taking the quick follow-up here. I just wanted to touch on Renaissance one more time and ask when we might hear a little bit more about the differentiation as it relates to the treatment process. Have you provided any color on the number of E. three cycles and how editing efficiency of the cash flow the enzyme may allow you to be more efficient in manufacturing the process.

Gilmore O’Neill

Thanks very much, Jack. So I'm going to ask Basil to cut.

Baisong Mei

Yes, then thank you, Al. We are, but we have not share specifics about the number of cycling the freezes when I mentioned before, Wes, since I joined, and we actually work together with our internal as well as a free seats, extra external freezes expert who actually have critical amendment to improve the process cycle and also provide assistance to study sites two for the PC cycle, which is significant because it's a reduced the patient burden is a smoother manufacturing process. We are very pleased to see the progress in that front. And just to add on that, we are hoping this clinical experience will be very much helpful for commercial and Dubai.

Operator

Mani Foroohar from Leerink Partners.

Mani Foroohar

Thank you for taking a follow-up question kind of similar to the last question. You've previously talked about optimizing the vein-to-vein process. Would you be able to walk us through how when still could provide advantages from either both operational and logistics perspective? Thank you.

Gilmore O’Neill

So based on I'm going to ask, I'll ask baseline to address that.

Baisong Mei

Yes. Yes, heavy heavy to our we are over these process really working together that too, with the study size to optimize these process and that coming from multiple factors. Some of the factors just mentioned above Reese's. The other factor is the logistics, and we provide support on that.
The first back is HE patient condition, as we know that we are treating this severe sickle cell disease and before that ran into a treatment that can have multiple BOE. per year. And that also can impact the vein-to-vein time.

Caren Deardorf

Manny, this is Karen. I would just add that again, in the fast follower position, one of the things it gives us the opportunity to do is to really understand as the first two therapies are commercialized, what's working, what's not working, what they need to see differently and really making sure that it is tough sets ourselves up as the partner of choice. And so we're working very hard on that, and we'll certainly talk about that at a later time.

Mani Foroohar

Great. Thanks, Dmitry, for your questions.

Operator

Ladies and gentlemen, this concludes today's call. Thank you once again for your participation. You may now disconnect.