Ladies and gentlemen, thank you for standing by and welcome to Sangamo Therapeutics Q3 2020 Webcast. At this time, all participants are in a listen-only mode. 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, Aron Feingold. Madam, please go ahead.

Good afternoon and thank you for joining us today. With me this afternoon on this call are several members of the Sangamo 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 R&D pipeline; 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 timelines for Sangamo and our collaborators to conduct clinical trials and share clinical data and the potential for these data to demonstrate clinical benefits to patients; the potential to use certain technologies to develop our therapies, our collaboration strategy and the potential to earn milestone payments and royalties from our collaborations and the timing of receiving such payments and royalties; plans and timelines for building and opening manufacturing facilities, the effects of the evolving COVID-19 pandemic; our expectations regarding our financial performance and resources and other statements that are not historical facts. 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 measure and reconciliations of GAAP to the non-GAAP measure discussed on this call are included in today’s press release, which is available on our website. Now, I would like to turn over the call to our CEO, Sandy Macrae.

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 in clinical execution and we are optimistic of our plans to continue to dose patients and initiate new trials. We also completed our research activities associated with our ALS collaboration with Pfizer and are continuing to move our research projects with Biogen and Novartis forward and are progressing our work with other partners. Pfizer has dosed the first participant in the Phase 3 AFFINE study of giroctocogene fitelparvovec or SB-525, our investigational gene therapy for hemophilia-A patients. This event triggered a $13 million milestone achievement for Sangamo, which we expect to receive in the current quarter further strengthening our cash position. Pfizer previously communicated that they expect pivotal data readout from the AFFINE study in 2022. During the mid-September Investor Day, Pfizer provided an update from the Phase 1/2 Alta study showing encouraging data regarding 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 Sangamo believe that these data support the potentially differentiated hemophilia-A gene therapy product candidate. In August and September, in close collaboration with principal investigators, monitoring safe conditions for patients within the context of COVID-19, Sangamo dosed the first two patients in the Phase 1/2 STAR study evaluating ST-920 gene therapy in Fabry disease. The first cohort of this study is now complete. An enrollment is ongoing for cohort 2. 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 1/2 clinical study evaluating CAR regulatory T cell…

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 partnered and wholly-owned programs. Pfizer dosed the first patient in the AFFINE study of giroctocogene fitelparvovec or SB-525, our first acid in a registrational trial. AFFINE is a global Phase 3 open-label multi-center single arm study evaluating the efficacy and safety of SB-525 in patients with moderately severe to severe hemophilia-A. The primary endpoint is annualized bleeding rates, or ABR to 12 months following treatment. This will be compared to ABR while in Factor VIII replacement Therapy collected in the Phase 3 bleeding study, which will provide a baseline for Phase 3 study participants. The secondary endpoints include Factor VIII activity level after the onset of steady state over 12 months. Participants will be analyzed throughout the 5-year study period following the single infusion to further assess durability of efficacy and safety. Pfizer shared updated Phase 1/2 data at a Pfizer investor event in September, which demonstrated that SB-525 was generally well tolerated. Each of the 5 patients in the high dose cohort sustained a clinically meaningful level of Factor VIII 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 ALTA study in the next few months, when all 5 patients in the 3e13 vector genomes per kilogram dose cohort have been followed for at least 1 year. We have dosed the first two patients comprising the first cohort in the Phase 1/2 STAR study evaluating ST-920 in Fabry disease. The goal of this gene therapy candidate is to provide a predictable and durable expression of…

Thank you, Bettina and good afternoon everyone. We are pleased to share our financial results for the third quarter of 2020. We reported a net loss of $1.6 million or $0.01 per share compared to a net loss of $27.3 million or $0.24 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 SB-525, our hemophilia-A candidate partnered with Pfizer and a $5 million milestone achieved for our C9orf72 collaboration with Pfizer. Turning to expenses, non-GAAP operating expenses, which exclude 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 BLA for SB-525 for Hemophilia-A. Turning to 2020 full year guidance, we are 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 will now turn it back to Sandy for closing remarks.

Thank you, Sung. We are focused on clinical execution and building momentum as we adapt to the conditions of COVID-19 and head towards the end of the year. We are 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 have put Sangamo in a strong position to achieve several important milestones and catalysts heading into next year. We believe we remain on track for AAV manufacturing facility in Brisbane to be operational at the end of this year and there are cell therapy facilities in Brisbane and Velban to be operational by year end 2021. We anticipate continued enrollment in Pfizer’s Phase 3 AFFINE study with a pivotal data readout expected by Pfizer in 2022 and also expect 1-year and 2-year Phase 1/2 data presentations over the next year and a half. We expect continued enrollment in the Phase 1/2 STAR study and the data readout towards the end of 2021. Sanofi has guided that the first data readout from the Phase 1/2 sickle cell disease study is expected next year. We anticipate presenting follow-up ST-400 beta-thalassemia data at the same time. And lastly, we expect that clinical trial initiations of the Phase 1/2 first-in-human CAR-T reg study will occur in 2021. And KITE expects that it’s study of allogeneic anti-CD19 CAR-T product candidate, KITE-037 will also commence in 2021. We look forward to delivering on these milestones in the coming year. Operator, please open the line for questions.

Certainly. [Operator Instructions] And our first question comes from the line of Geoff Meacham with Bank of America.

Hey, guys. Thanks for the question and congrats on all the pipeline progress. I had a couple on the first one is on the hemophilia-A study just with the 12 month analyze bleed rate endpoint, I want to kind of get your feedback on what FDA is, is looking for just relative to 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 CAR-T regs just wanted to maybe give us a little bit more context for how you see that differentiating and maybe what successes you see what the best probability of success, you see, and for example, solid tumors versus the liquid tumor cells? Thank you.

Thank you for your questions. On the first one around hemophilia-A, we are limited in what we can see, because this is known Pfizer sounds, we are so pleased with their progress into Phase 3, 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 CAR-T regs, I’m going to pass over to Jason who is a real expert in this area. And Jason, can you talk to us about how you see your CAR-T regs strategy?

Thanks, Sandy. And thanks for the question. So first, I’ll start off 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-in-human CAR-T regs study, TX200. We’re leaders in this field. And we’re developing and refining our understanding of CAR-T regs biology and rapidly advancing our ability to engineer and manufacture that T- cell. And our TX200 study will be the first-in-human 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-A2 negative patient will receive an HLA-A2 positive kidney transplant. And our therapy is comprised of T regs that are engineered to recognize A2 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 CAR-T regs. And this study will be important for us to understand the safety and efficacy in 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.

Okay, great. Thank you.

Thank you. Our next question comes from the line of Maury Raycroft with Jefferies.

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?

Maury, thank you very much for your question, we are very pleased with the progress of the T reg for HLA-A2 mismatch. We’re very pleased with the approval from the regulatory authorities, we call for dis epic where COVID is in is impacting the hospitals, we will go to 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.

Alright. And from a manufacturing and process development standpoint, is that all figured out or is there any other perspective you can provide on that?

One of the reasons that we acquired TxCell in 2018 was their understanding of how to look after T regs, which is different from how people look after T-cells. And so we’re very pleased with the progress that they’ve made.

Got it. Okay. And then the other question I had was just on the Prion 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.

Jason, can you hear me talk about Prions and what we think of them?

Sure, Sandy, thank you. The Prion program is in preclinical development. So I think, we’ll be looking forward to sharing updates at the appropriate time. I think that there is a great opportunity there 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 what our partners see in our platform. And the Prion program is get another example of that, and we’ll be excited to, to talk about it as we move forward. And we have done some of the initial work with the Broad Institute in Boston. And they have a real deep – the group we are working with has a real deep expertise in this. And so we have that biological expertise to match with our technological expertise.

Got it. Thank you for the perspective. Thanks for taking the questions.

Thank you, Maury.

Thank you. And our next question comes from the line of Jim Burchinal with Wells Fargo.

Hi, thanks for taking my question. This is a Yanan on for Jim, some perhaps a question on the T reg program as well. It – could you confirm whether this is a gene additive product? Because I think it’s autologous. So, it – the obviously the HLA-A2 is put in with a gene audition approach. So if you can comment on whether it’s a gene edited product, and then for your future product, the allogeneic regulatory CAR T-cells. How do you see the issue of persistence? I guess it’s for this program, TX200, 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 allogeneic programs? Thanks.

So, thank you for your question. They are very sensible scientific questions. And let me try and lay out the path that we have chosen which is to start with autologous so as 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 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 we can either develop allogeneics, T regs by editing down from healthy donor volunteers, or by studying IPSCs and other forms of stem cells to develop them up to be T regs. So this is really at the very cutting edge of regulatory cellular science and we are lucky to have as many options as possible to take us into those areas.

Got it. And a quick follow-up in TX200 is there a gene editing component?

TX200 is an autologous form and therefore it doesn’t have gene editing component. Jason, I am correct on that.

Yes, that’s correct. TX200, we introduced the CAR with a lentivirus for that first program, but obviously, our platform, one of the assets we have at Sangamo is our platform’s ability to do genomic engineering and the T regs for our future products.

And that was why the merge of Sangamo and TxCell was so sensible. They brought the T reg experience and we brought editing that they needed to take the platform forward.

Got it. Then maybe a question on zinc finger transcription factors, just wondering about the origin of the transcription factor, whether it is fully human, or is there any synthetic component in those transcription factors? And how do you think – how should we think about immunogenicity? Thanks.

Jason, do you want to have a go on that please?

Sure. 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 specific sequence in the genome and by nature of the fact that they are fully human we expect that the immunogenicity should be inherently lower. And we haven’t made any observations to-date to suggest that immunogenicity could be a problem.

Great. Thank you so much for taking the questions and congrats on our progress.

Thank you very much.

Thank you. And our next question comes from the line of Gena Wang with Barclays.

Thank you for taking my questions. I have three questions. Now, first is regarding hemophilia-A, just wondering, certainly I know this is already tech transferred to Pfizer given the [indiscernible] experience Phase 1/2 data did not – or Phase 3 data did not have quite difference from the Phase 1/2. So any thoughts you can share with us regarding your Phase 1/2 data and regarding also the Phase 3 data and one hypothesis was the manufacturing part. In essence, dose manufacturing Phase 1/2 is from your side and now we will transfer to the Pfizer, any thoughts you can help us understand in terms of our potential predictability from Phase 1/2 data to Phase 3? And then the second question is regarding Pfizer Partner Program for ALS, zinc finger 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 Fabry program, you already enrolled second cohort and what will be the determination that you think you will reach the optimal cohort that you can extend the cohort regarding the biomarker data like a plasma lyso-Gb3 as a reference, Avro showed 30% to 40% further reduction versus the baseline ERT. Any thoughts you have regarding what is your thought in order to determine the optimal dose?

So Gena, I am afraid I may disappoint you in my answers. The Pfizer – the Phase 2 of the Alta study and the progression of this with Pfizer is really in their hands to talk about and they announced or the abstracts for ASH were announced today. And so they will be talking about that at ASH and so I would guide you to wait for those results. The convertibility between Phase 2 and Phase 3, really we mustn’t see anything until we see the Phase 3 results. As regards the C9orf, 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 Fabry, I am going to pass on to Bettina. Bettina, can you talk about Fabry? And what we are looking for and what this study will measure?

Absolutely. So, yes, thank you for the question. So as I have mentioned earlier, we dose 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 α-Gal A 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 important parameters. But as you point out, we are also looking at substrates, Gb3 and lyso-Gb3 these are the basis of some of the challenges in terms of morbidity in patients with Fabry. So, we are 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 involved in the decisions as we move on to escalate, goes to the optimal dose. Think of this is with the totality of the data and the data that we are monitoring from other studies with other products, of course as well that we will be making our ultimate decisions on the dose that we bring forward in our cohort expansion.

Okay, thank you.

Thank you very much for your question.

Thank you. Our next question comes from the line of Eric Joseph with JPMorgan.

Hi, thanks for taking the question. Maybe just perhaps a strategic one, I am 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 forward to the clinic over the next 12 to 18 months, relative to I guess what we have seen over the past year, a fair amount of leveraging up the platform through partnering activities? Thanks.

Thank you for your question, Eric. Mark, this feels like one for you, please.

Hi, Eric. Thanks for the question. So if you take a look at the deals that we have done, including those most recently, really the driver of that is twofold. And in some cases, they come to us obviously in [indiscernible] and the couple of the candidates that we had progressing towards the end, but they came to us with a [indiscernible] expanding the number of CMS targets that we weren’t otherwise considering. So we looked at that as being really an extension of our potential pipeline. But the second way we sort of take a look at partnerships is whether they are bringing a specific expertise and the resources necessary to accelerate the products assuming they are 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 to become a genomics 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 what you have heard in terms of TX200 as well as the follow-on programs that we are intending to take forward into the clinic for our CAR-T reg programs.

Got it. I guess in addition to T reg, are there certain disease states or targets that you have essentially walled off and are prioritizing for internal development or?

We have not disclosed that. I mean, we are taking a look at, obviously, we have got interest in the autoimmune space as we have talked about, with our CAR-T reg programs and Jason touched on earlier. Naturally, we are working heavily within the CNS area with our partners in Biogen and our partnership with Novartis. And so, there maybe targets that we choose in that particular space, but we have not made any decisions on that as yet. We don’t believe you can really speak here and then go after it we need to let the science drive us towards things that we believe are important to develop for patients.

Got it. That’s helpful. Thanks for taking the question.

Sure. Pleasure.

Thank you. And our next question comes from the line of Ritu Baral with Cowen.

Hi, guys. Thank you for taking the question. This is Laila on for Ritu. Just two quick questions from me. First, on the STAR Fabry study, are you still seeing impacts from COVID on enrollment? I know you have 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, 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 any color on this would be helpful? Thank you.

Thank you for your question. We haven’t – we or Pfizer haven’t commented on the ALS program and when the monsoons will come. I would say again, I would reiterate what I said in the call script, it’s a remarkable piece of science to be able to suppress the transcription of one [indiscernible] and leave the other one untouched is why Pfizer came to us and we have achieved what they requested of us and it’s moved 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 am going to refer you to Bettina who will answer that question?

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. During the pandemic, we have 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 dosing of the first two patients to keep going with our enrollment despite the pandemic at this stage where the things we have put in place from an operational perspective with home visits and with virtual assistants have really helped us make sure that, we together with the sites manage to guide the patients through the enrollment and screening procedures to ultimately get us to dosing and then follow-up.

Got it. Okay, thank you for the color.

I am very pleased with the way that clinical team have navigated. We feel we have a responsibility to do this well to make sure that the patient comes first whether they are being treated for COVID in which case we should not be taking up doctors’ times. But we have been ready to dose as soon as the window was open. And I think Bettina and her team have done remarkable job to get us there.

Got it. thank you for the color.

Thank you. Our next question comes from the line of the Nicole Germino with Truist.

Good evening, everyone. Hi, thanks for taking my question. On Fabry, given the competition in the space, what is it more specifically about Sangamo’s AAV tested or the promoter that may get differentiated and better than your peers? And how have you made that determination? And then does – how does ST-920 impact the renal and cardiac tissue?

So, let me take that one. We are encouraged by the data we have seen from AV6 in hemophilia-A. But until we dosed 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 achieved supramaximal dosing and supramaximal effect. And in the animal dosing that we recently published, we are able to show benefit to both the heart and the kidney. But I am just prudent in telling people to wait and let’s see what the clinical results are. We are guiding that will show the clinical results towards the end of next year when the dose escalation phase is completed.

Okay, great. Thank you so much.

Thank you. And our next question comes from the line of Evan Wang with Guggenheim Securities.

Hi, guys. Thanks for taking my question. I had two, one on heme-A and other on the allo CAR-T program, on heme-A what’s the plan going forward to share data on the Phase 1/2. I know you will have the update at ASH, but are there any specific time points prior to complete picture of the data and without requiring – all patients require across that time point and on the allo CAR-T program, we have seen some data, early data from other allo programs from allogene and CRISPR. Any thoughts on the initial data generated so far and any kind of learnings you can take, especially given some of the deaths we have seen in those trials?

So, let me do the heme-A and then pass this to Jason to comment on allo. So I am going to say again [indiscernible] to Pfizer will – are in control of the release of data on hemophilia-A. There is an abstract that’s been accepted for ASH and they will use for conference schedule in the future to continue to demonstrate the benefit to patients of reduced absent bleeding events and no requirement for factor. Jason, can you talk about the allogeneic question, please?

Sure. Thank you, Sandy. So I believe that you are referring to allo CAR-T programs in oncology and we have our partnership as you know with KITE Gilead to support their allogeneic CAR-T programs. KITE has guided that we are expecting to begin the studies on their allogeneic CD-19 targeted CAR-T therapy next year and we believe that the data that we have seen so far from others are consistent with these therapies having real therapeutic potential and we are excited about it and we are excited to see KITE get the therapies into patients.

Okay, thank you.

Thank you. I will now turn the call back over to Head of Corporate Communications, Aron Feingold for any further remarks.

Thank you once again for joining us today and for your questions. We look forward to keeping you updated on our future developments.

Ladies and gentlemen, this concludes today’s conference call. Thank you for participating and you may now disconnect.