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Sangamo Therapeutics Inc (SGMO -0.85%)
Q3Â 2020 Earnings Call
Nov 4, 2020, 5:00 p.m. ET
Contents:
- Prepared Remarks
- Questions and Answers
- Call Participants
Prepared Remarks:
Operator
Ladies and gentlemen, thank you for standing by, and welcome to Sangamo Therapeutics Inc Q3 2020 webcast. [Operator Instructions] After the speaker presentation there will be a question and answer session. [Operator Instructions]
I would now like to hand the conference over to your host head of Corporate Communications McDavid Stilwell, please go ahead.
McDavid Stilwell -- Head of Corporate Communications
Good afternoon. And thank you for joining us today. With me this afternoon on this call are several members of the same demo executive leadership team, including Sandy Macrae, Chief Executive Officer. Sung Lee Chief Financial Officer, Mark McClung, Chief Business Officer, Jason Fontenot, Interim Head of Research and Bettina Cockroft Chief Medical Officer. Slides from our corporate presentation can be found at our website, sangamo.com, under the Investors and Media section in the Events and Presentations page. This call includes forward-looking statements regarding Sangamo's current expectations. These statements include, but are not limited to, statements relating to our pipeline of genomic medicine product candidates; our ability to develop, obtain regulatory approvals for and commercialize therapies to treat certain diseases and the timing, availability and cost of such therapies; plans and time lines for Sangamo and to conduct clinical trials and share clinical data and the potential for data to demonstrate clinical benefit to patients; the potential to use certain technologies to develop AR therapies, our collaboration strategy and the potential to earn fees, milestone payments and royalties from our collaboration; plans and time lines for building and opening manufacturing facilities, the effects of the evolving COVID-19 pandemic; the anticipated benefits of our organizational changes; our expectations regarding our financial performance and resources and other statements that are not historical fact. Actual results may differ substantially from what we discuss today.
In addition, these statements are not guarantees of future performance and are subject to certain risks and uncertainties that are discussed in documents that we file with the securities and Exchange Commission, specifically in our quarterly report on Form 10-Q for the quarter ended September 30, 2020. The forward-looking statements stated today are made as of this date, and we undertake no duty to update such information, except as required under applicable law. On this call, we discuss a non-GAAP financial measure. We believe this measure is helpful in understanding our past financial performance and our potential future results. This is not meant to be considered in isolation or as a substitute for the comparable GAAP measure. The comparable GAAP measures and reconciliations of GAAP to the non-GAAP measures discussed on this call are included in today's press release, which is available on our website.
Now, I'd like to turn the call over to our CEO, Sandy Macrae.
Sandy Macrae -- Chief Executive Officer
Thank you, and good afternoon to everyone on the call. This quarter. We advanced our r&d activities as we continue to adapt to the conditions brought on by the evolving Covid-19 pandemic. We are moving forward on clinical execution. And we're optimistic we're plans to continue to doors patients and initiate new trials. We also completed our research activities associated with our als control collaboration with slide and are continuing to move our research projects with Biogen and Novartis forward and are progressing and work with our other partners. Pfizer has dosed the first participant in the phase three of fine study of JIRA tokujin, fetal Purbeck or SP five to five or investigational gene therapy for Haemophilia a patients. This event triggered a $13 million milestone achievement for sango, which we expect to receive in the current quarter, further strengthening our cash position. Pfizer previously communicated that they expect a pivotal data readout from the final study in 2022. During the mid September investor day, Pfizer provided an update from the phase one to alter study showing encouraging data garnering tolerability clinically meaningful factor levels, bleeding rates and factor use in the highest dose cohort up to 85 weeks in the longest treated patients. Pfizer and sanken will believe that these data support a potentially differentiated Haemophilia a gene therapy product candidate in August and September, in close collaboration with principal investigators monitoring safe conditions for patients with the within the context of COVID-19 single dose the first two patients in the phase one two star study evaluating STM 20 gene therapy in Fabry disease. Those are the first cohort of this study is no complete. And enrollment is ongoing for Cohort Two.
We expect to share data on this study by the end of next year. During the quarter, we received additional regulatory approvals for the first in human Phase One Two clinical study evaluating car regulatory T cell or car t reg, candidate tx 200 and kidney transplantation. We believe we are on track to initiate the study next year. initiating the study may allow us to be the first company to explore the potential of car T cells in humans. We are hopeful that this will provide broader proof of concept for genetically engineered cell therapy using T rex beyond transplantation. We intend to further evaluate current T Rex, including zinc finger nucleases edited allergenic t reg therapies in autoimmune diseases with high unmet medical need. Also, this quarter we completed our research activities associated with our ongoing Pfizer collaboration to develop gene regulation therapy. Using zinc finger technology for the treatment of C nine or 72 related als in this program are safe finger proteins are designed to selectively target disease alio repeats a remarkable demonstration. Yet another way our versatile technology may be able to have a disease modifying impact on challenging CNS diseases. We recently earned a 5 million milestone payment from Pfizer so stay with this program, which we expect to receive later this quarter. It is a testament to our r&d momentum. We look forward to continue to work closely with Pfizer to support their research and development in this program.
With that, I will turn the call over to our chief medical officer Bettina, who will provide additional details on our clinical accomplishments.
Bettina Cockroft -- Senior Vice President and Chief Medical Officer
Good afternoon. As Sandy mentioned, our clinical operations have adapted to the challenges of the evolving covid 19 pandemic. And we are pleased with our progress in executing on our partner and wholly owned programs. Pfizer those the first patient in the assigned study of director gene fetal Popovic or SB five to five, our first acid in a registrational trial. A fine is a global phase three open label multicenter single arm study evaluating the efficacy and safety of SB five to five. in patients with moderately severe to severe Haemophilia a. The primary endpoint is annualized bleeding rates or APR through 12 months following treatment. This will be compared to ABR while in fact eight Replacement Therapy collected in the phase three lead and study, which will provide a baseline for phase three study participants. The secondary endpoints include factor eight activity level after the onset of steady state over 12 months. Participants will be analyzed throughout the five year study period following the single infusion to further assess your ability of efficacy and safety.
Five has shared updated phase one two data at a Pfizer investor events in September, which demonstrated that sb 525 was generally well tolerated. Each of the five patients in the high dose cohort sustained a clinically meaningful level effect great activity without leads or the need for prophylactic factor up to 85 weeks for the longest treated patient. Both companies are encouraged by these results and plan to present further follow up data from the altia study in the next few months. When all five patients in the 313 vector genomes per kilogram dose cohort have been followed for at least one year. We have those the first two patients comprising the first cohort in the phase one to start study evaluating SPSS 20 in Fabry disease. The goal of this gene therapy candidate is to provide a predictable and durable expression of the alpha gal a enzyme, which is deficient and Fabry disease due to mutations in the GLA gene, resulting in the accumulation of the substrates GP three, and it's soluble derivative lyso Gp three. This can cause challenging symptoms and morbidities including impaired renal and cardiac function, pain and gastrointestinal symptoms. The star trial is a multicenter open label those ranging study evaluating the safety and tolerability of St. 920 and classical Fabry patients 18 years and older study participants will receive a single intravenous infusion of St. 920, followed by one year of observation and monitoring of clinical endpoints, such as alpha gelei activity and assessment of GB three and laser GB three levels. A long term follow up study will allow patients to be monitored for an additional four years. enrollment for the second cohort is ongoing. We expect that data will be shared toward the end of 2021 after we have identified adults code for cohort expansion.
We believe that sc 920 offers a potentially differentiated treatment for Fabry disease, with the potential to deliver efficacy with preserved renal function and reduced cardiac morbidity and neuropathy. preclinical studies evaluating STI 20 demonstrated strong expression of alpha Galle and G v3 substrate reduction across tissue types. As delivered directed gene therapy, Sc 920 is delivered by a one time intravenous infusion that does not require any preconditioning regimen for patients. We are also working closely with our oncology collaborator Kate Gilliard company, as it advances code 037 and allergenic and PCV 19 Carty therapy into clinical trial tight expects to submit an investigational new drug application by the end of 2020 and to initiate a clinical trial evaluating kite 037 in 2021.Throughout the third quarter, we have continued to receive additional regulatory approvals that support the phase one to steadfast clinical study evaluating the first inhuman car t reg cell therapy tx 200 in HLA to mismatched renal transplantation, we expect to initiate the study next year. The goal for this study is the prevention of transplant rejection through the engineering of T regs to express an HLA to kemmerich antigen receptor or car, allowing them to localize to the renal graft and activate upon recognition of the HLA to antigen. The Carty regs may prevent immune mediated rejection through the inhibition and modulation of inflammatory immune cells and the release of anti inflammatory cytokines to induce a tolerogenic environment within the preclinical data supporting the steadfast study presented last month showed that the TX 200 HLA to Carty regs efficiently prevented rejection in both graft versus host disease and skin transplantation model.
They were also shown to be safe and well tolerated in our in vivo studies. Similar to other genetically engineered cell therapy approaches, patients will undergo a Luca phoresis procedure from which their t reg cells will be isolated and engineered then cryopreserved. The HLA to negative patient will subsequently undergo transplantation surgery and following a recovery period will receive their personalized pX 200 drug candidate.As a result of this detailed process, we expect those patients will occur several months after their enrollment. recent publications show that the regulatory cell therapy space is gaining momentum and excitement in the scientific community. In particular, the one study a large international clinical study gathering seven investigator led trials across five countries showed that immune regulatory cell therapy as a whole were safe, and that immune cell therapy is a potentially useful therapeutic approach in renal transplant recipients allowing immune cell composition restoration to normal healthy level of minimization of the burden of general immune suppression. This is very promising and supports our plan to evaluate Carty regs in renal transplant patient populations.
Will now turn the call over to some for an overview of the financial results.
Sung Lee -- Executive Vice President and Chief Financial Officer
Thank you, Bettina and good afternoon everyone. We're pleased to share our financial results for the third quarter of 2020. We reported a net loss of $1.6 million, or one cent per share compared to a net loss of $27.3 million or 24 cents per share for the same period in 2019. total revenues were $57.8 million, compared to $22 million for the same period in 2019. The increase was primarily attributable to a $30 million milestone achieved. For SP five to five or Haemophilia, a candidate partnered with Pfizer, and a $5 million milestone achieved for our C nine or 72 collaboration with Pfizer. Turning to expensive non GAAP operating expenses, which include stock based compensation expense were $54.8 million, compared to $46.5 million for the same period in 2019. The increase in operating expense reflects our headcount growth and facilities expansion to support the advancement of our therapeutic pipeline, and manufacturing capabilities.
These increases were partially offset by a decrease in clinical and manufacturing supply expenses. Moving to the balance sheet, we ended the quarter with $695 million in cash, cash equivalents and marketable securities. This balance includes the $75 million upfront license fee payment received from Novartis. Additionally, in the current quarter, we expect to receive the $35 million milestone payments from Pfizer mentioned earlier. We believe our balance sheet remains strong and will allow us to reach several important r&d milestones, including the potential filing of the Vla for sp 525. For hemophilia a. Turning to 2024 year guides, we're updating our financial guidance for non GAAP operating expenses, which exclude estimated stock compensation expense of $25 million from an estimated range of 210 million to $225 million to now be in the estimated range of 210 million to $220 million.
I'll now turn it back to Sandy for closing remarks.
Sandy Macrae -- Chief Executive Officer
Thank you. So we're focused on clinical execution and building momentum. As we adapt to the conditions of COVID-19 and head toward the end of the year. We're pleased with our progress in clinical operations and with our partner programs. Our strong balance sheet enables us to advance our r&d pipeline. We believe these accomplishments are put single in a strong position to achieve several important milestones and catalysts heading into next year. We believe we remain on track for a V manufacturing facility in Brisbane to be operation at the end of this year, and offeror cell therapy facilities in Brisbane and football to be operational by year end 2021. We anticipate continued enrollment in Pfizer's phase three a fine study with a pivotal data readout expected by Pfizer in 2022. And also expect one year and two year phase one two data presentations over the next year and a half. We expect continued enrollment in the phase one two star study and the data read data readout toward the end of 2021.
Santa Fe has guided that the first data readout from the phase one to sickle cell disease study is expected next year. We anticipate presenting pull up sp 400 beta thalassemia data at the same time. And lastly, we expect the clinical trial initiation. So the phase one two first inhuman Carty reg status study will occur in 2021. And Kate expects that this study of allogeneic anti cd 19 Carty product candidate count or three seven will also commence in 2021. We look forward to delivering on these milestones in the coming year.
Operator, please open the line for questions.
Questions and Answers:
Operator
[Operator Instructions] Our first question comes from the line of Geoff Meacham With Bank of America.
Geoff Meacham -- Bank of America -- Analyst
Hey guys, thanks for the question and congrats on all the pipeline progress. I had a couple on the first one is on the Haemophilia a study just with the 12 month analyze bleed rate endpoint, I want to kind of get your feedback on on what FDA is, is looking for just relative to, you know, the feedback that biomarin received. And that's obviously subject of a lot of investor conversations of late. And then the second question is just a broader one on the strategy for the car t regs just wanted to maybe give us a little bit more, you know, context for, you know, how you see that differentiating and maybe what successes you see what the best probability of success, you see, and, and for example, solid tumors versus the liquid tumor say. Thank you.
Sandy Macrae -- Chief Executive Officer
Thank you for your questions. On the first one around Haemophilia a, we are limited in what we can see, because this is known Pfizer sounds, we are so pleased with their progress into phase three, with their enthusiasm for the program, and that all the way up and down their organization right up to their CEO, how valuable they see this asset for them. The you can be assured that they will be having regular conversations with the regulatory authorities. And I'm certain that Pfizer will know how to navigate that landscape. As regards to T regs, I'm going to pass over to Jason who is a real expert in this area.
Geoff Meacham -- Bank of America -- Analyst
And Jason, can you talk to us about how you see your T reg strategy?
Jason Fontenot -- Senior Vice President of Cell Therapy; Interim Head of Research
Thanks, Sandy. And thanks for the question. Um, so first, I'll start off by by pointing out that our programs and regulatory T cells, engineered regulatory T cells are directed in the autoimmune and inflammatory disease space, these are drugs for cancer. So we're really excited about our engineered regulatory T cell platform and the programs that we're bringing forward. We're excited about the progress and regulatory approvals we've received so far. And about our first inhuman. Carty reg study, Tx 200. We're leaders in this field. And, you know, we're developing and refining our understanding of cartoon t reg biology and rapidly advancing our ability to engineer and manufacture that cell. And our tx 200 study will be the first inhuman test of engineered regulatory T cells of car t regs, and this will be further demonstration of our leadership.
The goal with that study is the prevention of renal transplant rejection in the setting of an MHC mismatch transplant. So this is a setting where and HLA a two negative patient will receive an HLA a two positive kidney transplant. And our therapy is comprised of T regs that are engineered to recognize the a two antigen through a Comerica antigen receptor and that car that will drive the accumulation and activation of the T regs in the renal graft and suppress the rejection of the graft by the patient's immune system. And what's important about that study is that as I mentioned, this is going to be the first test of this therapeutic hypothesis around core t regs. And this study will be important for us to understand the safety and efficacy and the therapeutic potential of car t regs. And we'll be informing programs that we're actively pursuing enlarger autoimmune and inflammatory indications such as multiple sclerosis and Crohn's disease.
Geoff Meacham -- Bank of America -- Analyst
Okay, great. Thank you.
Operator
Thank you. Our next question comes from the line of Maurice Raycroft with Jefferies.
Maurice Raycroft -- Jefferies -- Analyst
Hi, Everyone, thanks for taking my questions. I had one on the T reg program as well. So I guess for getting the getting that study started. Can you talk more about what factors have led to pushing the study start to 2021? Is it due to COVID or the autologous cell manufacturing process or anything else that you can comment on? And can you talk more about what else needs to be completed before starting the study?
Sandy Macrae -- Chief Executive Officer
Maurice, thank you very much for your question. We are we're very pleased with the progress of the T reg for HLA to mismatch. We're very pleased with the approval From the regulatory authorities, we call for dis epic where COVID is in. In is impacting the hospitals, we will go to the the laboratories, the manufacturing. So there's a general COVID impact, but we are confident that we'll be able to move forward with this program.
Maurice Raycroft -- Jefferies -- Analyst
And from a manufacturing and process development standpoint, is that all figured out? Or is there any other perspective you can provide on that?
Jason Fontenot -- Senior Vice President of Cell Therapy; Interim Head of Research
One of the reasons that we acquired t excel in 2018, was their understanding of how to look after T Rex, which is different from how people look after T cells. And so we're very pleased with the progress that they've made.
Maurice Raycroft -- Jefferies -- Analyst
Got it. Okay. And then the other question I had was just on the preamp program that you guys have, which is sort of under the radar, I'm just wondering if you can provide a status update on that program, any timeline, update on that program, and then maybe talk about the strategic importance of that one, as well.
Sandy Macrae -- Chief Executive Officer
Jason, can you hear me talk about prions and what we think of them?
Jason Fontenot -- Senior Vice President of Cell Therapy; Interim Head of Research
Sure, Sandy, thank you. Um, you know, the prion program is in preclinical development. So I think, you know, we'll be looking forward to sharing updates at the appropriate time. I think that there is a great opportunity there to to demonstrate the power of our platform, similar to the approaches that we're taking with our partners in CNS, both Biogen and Novartis. These are both of these partnerships are driven by by what our partners see in our platform. And the prion program is yet to get another example of that, and we'll be excited to, to talk about it as we move forward. And we've done some of the initial work with the Broad Institute in Boston. And they have a real deep x the group we're working with as a real deep expertise in this. And so we have that biological expertise to match with our technological expertise.
Maurice Raycroft -- Jefferies -- Analyst
Got it. Thank you for the perspective. Thanks for taking the questions.
Jason Fontenot -- Senior Vice President of Cell Therapy; Interim Head of Research
Thank you, Marie.
Operator
Thank you. Next question comes from the line of Jim Burchinal with Wells Fargo.
Jim Burchinal -- Wells Fargo. -- Analyst
Hi, thanks for taking my question. This is a yen and on for Tim, some perhaps a question on the T reg program as as well. It, Could you confirm whether this is a gene additive product? Because I think it's autologous. So, it you know, the obviously the HLA two is put in with a gene audition approach. So if if you can comment on whether it's a gene edited product, and then for your, you know, future product, the genetic regulatory car T cells. How do you see the issue of persistence? I guess it's for this program, GTX 200, as well. How do you see the issue of persistence? And for renal transplant, would you require long term persistence? And would you explore repeated dosing for your animal genetic programs? Thanks.
Jason Fontenot -- Senior Vice President of Cell Therapy; Interim Head of Research
So thank you for your question. They they're very sensible, scientific questions. And let me try and lay out the path that we've chosen, which is to start with a topic of sorts, we can understand the effectiveness of T regs. And one of the advantages of renal transplant is that the transplanted kidney can be biopsied because it's it's implanted close to the surface. So, we can look at things like persistence which as you can see is an important feature that would be required of any good treatment, we will gradually switch to allergenic and, and we can either develop our genetics, t regs by editing down from healthy donor volunteers, or by studying IPS, C's and other forms of stem cells to develop them up to be T Rex. So this is really at the very cutting edge of regulatory cellular science and we are lucky to have many options as possible to take us into those areas.
Jim Burchinal -- Wells Fargo. -- Analyst
Got it? And a quick follow up in tx 200. Is there a gene editing component?
Sandy Macrae -- Chief Executive Officer
Tx 200 isn't a tall Lucas form and therefore doesn't have gene editing component? Jason, I'm correct on that.
Jason Fontenot -- Senior Vice President of Cell Therapy; Interim Head of Research
Yes, that's correct. tx 200. We introduced the car with a lentivirus for that first, for that first program. But obviously, you know, our platform, one of the one of the assets we have etang. Mo is our platforms ability to do genomic engineering and the T rex are our future future products. And that was why the marriage of sanguine to excel was so sensible. They they brought the TV experience and we brought editing that they read, they needed to take the platform forward.
Jim Burchinal -- Wells Fargo. -- Analyst
Got it, then maybe a question on zinc finger transcription factors. Just wondering about the origin origin of the transcription transcription factor, whether it is Foley Schumann, or is there any synthetic component in those transcript transcription factors? And how do you think how should we think about immunogenicity? Thanks.
Jason Fontenot -- Senior Vice President of Cell Therapy; Interim Head of Research
Sure, so, so the base components of the zinc finger transcription factors are all human. Obviously, in order to direct those transcription factors to a desired sequence, we have to design a synthetic protein that is specific for a specific sequence in the genome. And, and by nature of the fact that they are fully human, we expect that the immunogenicity should, should be inherently lower. And we haven't made any observations today to suggest that immunogenicity can be a problem.
Jim Burchinal -- Wells Fargo. -- Analyst
Great, thank you so much for taking the question and congrats on your progress.
Sandy Macrae -- Chief Executive Officer
Thank you very much.
Operator
Thank you. And our next question comes from the line of Gena Wang with Barclays.
Gena Wang -- Barclays -- Analyst
Thank you for taking my questions. I have three questions. Now. First is regarding Haemophilia a. Just wondering, certainly I know this is already tech transferred to Pfizer given the ball milling experience, phase one two data did not three data dinner had quite different from the least one two. So you know, any thoughts you can share with us regarding your face one two data and regarding. you know, also the phase three data and one hypothesis was the manufacturing part. In essence, you know, this manufacturing Phase One, two is from your side. And now we'll transfer to the Pfizer in any thoughts you can help us understand in terms of our potential predict ability from phase one to data to phase three.
And then the second question is regarding Pfizer Partner Program, the ALS didn't single protein transcription gene regulation program. So wondering if you can share any color regarding the efficiency from this initial r&d study. And the last question is regarding the February program, you already enrolled second cohort and what will be their determination that you think you reach the optimal cohort that you can extend the cohort regarding the biomarker data like a plasma lecture with GD three, you know, as a reference, Avro showed 30 to 40% further reduction versus the baseline er T. Any thoughts you have regarding what is your boss in order to determine the optimal dose?
Jason Fontenot -- Senior Vice President of Cell Therapy; Interim Head of Research
I'm afraid I may disappoint you in my answers the visor the face two of the ultra study and the progression of of this with Pfizer is really in their hands, no to talk about. And the announced or the abstracts for ash were announced today. And so there'll be talking about that at ash and so I would go to to to wait for those results. The convertibility between phase two and phase three, really we we mustn't see anything until we see the three results. As regards the SR north. All I can tell you is Pfizer very pleased with the product that we produce for them. And they have transferred into their research organization and paid the necessary milestone. As regards fabri, I'm going to pass on to Bettina Bettina. Can you talk about fabri? And what we're looking for and what this study will measure?
Bettina Cockroft -- Senior Vice President and Chief Medical Officer
Absolutely. So, yes, thank you for the question. So as I've mentioned earlier, we don't the first cohort of two patients, and enrollment is ongoing. And the study goal really is to provide a predictable and durable expression of the alpha Galle enzyme, which is the enzyme deficient in Fabry disease due to mutations in the GLA gene. And so we are going to be measuring this parameter as one of the more more important parameters. But as you point out, we're also looking at substrates. GB, three and Lysa, GB, three, these are the basis of some of the challenges in terms of morbidity in patients with fabri. So we're going to be monitoring all of this data along with other data. Remember, this is first and foremost, the safety and tolerability study to start off with. And we have a safety monitoring committee that will be will be involved in the decisions as we move on to escalate, goes to the optimum dose, think it is, is with the totality of the data, and the data that we are monitoring from other studies with with other products, of course, as well that we will be making our ultimate decisions on the dollars that we bring forward in our cohort expansion.
Gena Wang -- Barclays -- Analyst
Okay, thank you.
Jason Fontenot -- Senior Vice President of Cell Therapy; Interim Head of Research
Thank you very much for your question.
Operator
Thank you. Our next question comes from the line of Eric Joseph with JPmorgan.
Eric Joseph -- JPmorgan -- Analyst
Hi, thanks for taking the question. Maybe just perhaps this cheated. One. I'm curious to get a sense of whether there are? Well, how we should be thinking about the potential for additional, largely wholly owned programs coming for the clinic over the next 12 to 18 months, relative to I guess what we've seen over the past year, a fair amount of, you know, leveraging up the platform through partnering activities.
Jason Fontenot -- Senior Vice President of Cell Therapy; Interim Head of Research
Next. Thank you for your question, Eric. Mark, this feels like one for you, please.
Mark McClung -- Executive Vice President and Chief Business Officer
Hi, Eric, thanks for the question. You know, so if you take a look at the, the deals that we've done, including those most recently, really the driver of that is is twofold. And in some cases, they've come to us on, you know, obviously in the the charging case intersect and in the couple of the candidates that we had progressing toward the the end. But they came to us with a view taxi, expanding the number of CMS targets that we weren't otherwise considering. So we looked at that as being really an extension of our, our potential pipeline. he second way we sort of took take a look at partnerships is whether they're bringing a specific expertise and the resources necessary to accelerate the products assuming they're successful to patients, and in that particular case, as well as the Novartis case. Both of those really fit those type of criteria. We fully intend and we to to become a genomic medicine company. And we are continually looking to advance wholly owned assets, which we would take into the clinic and I think a good example of that is is what you heard in terms of tx 200 as well as the follow on programs that we're intending to take forward into the clinic for our car t reg programs.
Eric Joseph -- JPmorgan -- Analyst
Got it I guess in addition to T reg, are there certain disease states over targets that you have essentially walled off and are prioritizing for internal development.
Mark McClung -- Executive Vice President and Chief Business Officer
We'd not disclose that. I mean, we're we're taking a look at, obviously, we've got interest in the autoimmune spaces, as we talked about, with her Carty read programs. And Jason touched on earlier. You know, naturally, we're working heavily within the CNS area with our partners in Biogen and partnership with Novartis. And so, you know, there may be targets that we choose in that particular space, but we've not made any decisions on that as yet. We don't believe you can really stick areas and go after it, we need to let the science drive us toward things that we believe are important to develop for patients.
Eric Joseph -- JPmorgan -- Analyst
God, that's a tough one. Thanks for taking the question.
Jason Fontenot -- Senior Vice President of Cell Therapy; Interim Head of Research
Sure.
Operator
Thank you. Our next question comes from the line of Ritu Baral with Cowen.
Ritu Baral -- Cowen -- Analyst
Hi, guys, thank you for taking the question. This is while on for two, just two quick questions from me. First, on the star fabri study, are you still seeing impacts from COVID on enrollment? I know you've completed the first cohort, but are all the sites up and running? And then as a quick follow up, regarding the Pfizer collaboration with the ALS program, you know, what are the next steps for the program? Specifically, what's the next opportunity for a milestone to reach is that contingent on them initiating a clinical trial? and unicolor on this would be helpful. Thank you.
Sandy Macrae -- Chief Executive Officer
Thank you for your question. We haven't we or Pfizer haven't commented on the LS program and when the mouse ones will come? I would say again, reiterate what I said in the call script. It's a remarkable piece of science to be able to suppress the transcription of one Leal and leave the other one, on touched is why Pfizer, Kim does and we have achieved what they requested of us, and it's move forward to them. So let's hope because it's such a dreadful disease, let's hope it gets to patients as soon as possible. For the Fabry disease, I'm going to refer you to Bettina who will answer that question?
Bettina Cockroft -- Senior Vice President and Chief Medical Officer
Yes, and thank you for that question. So on the clinical operations side, I have to say, we have an excellent clinical operations team working very hard on maintaining the relationship with all the sites on during the pandemic, we've also been able to initiate sites qualify other sites as we expand our footprint for the study. Of course, COVID has had an impact worldwide, some regions more than others, we are confident that we can keep going with following the enrollment and dozing of the first two patients to keep going with our enrollment, despite the pandemic at this stage where the the things we've put in place from an operational perspective with home visits with virtual assistants have really helped us make sure that but we, together with the sites manage to to guide the patients through the enrollment and screening procedures to ultimately get us to dosing. Very pleased. I'm very, very pleased with the way the clinical to have navigated. We feel we have a responsibility to do this. Well, to make sure that the patient comes first whether they're being treated for coffered in which case we should not be taking up doctors times. But we've been ready to doors as soon as the window was open. And I think Bettina and her team have done remarkable job together.
Ritu Baral -- Cowen -- Analyst
Thank you for the color.
Operator
Thank you. Our next question comes from the line of the Nicole Germino with Securities.
Nicole Germino -- Securities -- Analyst
Good evening, everyone. Hi, thanks for taking my question. On February, given the competition in this space, what is it more specifically about saying the most a decapitated or the promoter that make it differentiated and better than your peers? And how have you made that determination? And then does how does sc 920 impacts the renal and cardiac tissue?
Jason Fontenot -- Senior Vice President of Cell Therapy; Interim Head of Research
So let me take that one. We are encouraged by the data we've seen from AV six in hemophilia. But until we those patients with Fabry disease, we really can't and shouldn't comment on what it's going to look like. The animal data looks very encouraging. We see supramaximal dozing and supramaximal effect. And in the animal dosing that we recently published, we're able to show benefit to both the heart and the kidney. But I'm, I'm just prudent in telling people to wait and let's see what the clinical results are. Were guiding that will show the clinical results to RCM just next year when the Deus Ex escalation phase is completed.
Nicole Germino -- Securities -- Analyst
Okay, great. Thank you so much.
Operator
Thank you. Next question comes from the line of Boran Wang with Guggenheim securities.
Boran Wang -- Guggenheim securities -- Analyst
Hi, guys, thanks for taking my question. I had to one on PMA and other on the Albuquerque program on him a what's the plan going forward to share data on the Phase One, two, I know you'll have the updated app. But are there any specific time points provide a complete picture of the data and without requiring of all patients require cross that time point. And on the alto Carty program?
Sandy Macrae -- Chief Executive Officer
Yeah, we've seen some data, early data from other Allo programs from allergy and CRISPR. No any thoughts on the initial data generated so far, and any kind of learnings you can take, especially given some of the deaths we've seen in those trials? So let me do the humane and then passes to Jason to comment on aloe. So the I'm gonna say again, why piercing through Pfizer will are in control of the release of data on hemophilia. The there is an abstract has been accepted for ash and they will use whatever conference schedule in the future to continue to demonstrate the benefit to patients or reduce absent bleeding events and no requirement for factor. Jason, can you talk about the allergy alginate question, please.
Jason Fontenot -- Senior Vice President of Cell Therapy; Interim Head of Research
Sure. Thank you, Sandy. So I believe that you're referring to aloe Carty programs in in oncology and not we have a partnership with Gilead to support their allogeneic Carty programs. kite has has guided that we're expecting to begin the studies on on their alpha genetics cd 19 targeted Carty therapy next year. And we believe that the data that we've seen so far from from others are consistent with, with these with these therapies having real therapeutic potential and we're excited about it and and we're excited to see Kai get the get the therapies into patients.
Boran Wang -- Guggenheim securities -- Analyst
Okay, thank you.
Operator
Thank you. I will now turn the call back over to head of corporate communications. Sandy Macrae for any further remarks.
Sandy Macrae -- Chief Executive Officer
Thank you once again for joining us today and for your questions. We look forward to keeping you updated on our future developments.
Operator
[Operator Closing Remarks]
Duration: 49 minutes
Call participants:
McDavid Stilwell -- Head of Corporate Communications
Sandy Macrae -- Chief Executive Officer
Bettina Cockroft -- Senior Vice President and Chief Medical Officer
Sung Lee -- Executive Vice President and Chief Financial Officer
Jason Fontenot -- Senior Vice President of Cell Therapy; Interim Head of Research
Mark McClung -- Executive Vice President and Chief Business Officer
Geoff Meacham -- Bank of America -- Analyst
Maurice Raycroft -- Jefferies -- Analyst
Jim Burchinal -- Wells Fargo. -- Analyst
Gena Wang -- Barclays -- Analyst
Eric Joseph -- JPmorgan -- Analyst
Ritu Baral -- Cowen -- Analyst
Nicole Germino -- Securities -- Analyst
Boran Wang -- Guggenheim securities -- Analyst
