McDavid Stilwell - Vice President, Corporate Communications and Investor Relations Sandy Macrae - President and CEO Edward Conner - Senior Vice President and CMO Michael Holmes - Vice President, Research Curt Herberts - Senior Vice President and CBO Kathy Yi - Senior Vice President and CFO.

Jim Birchenough - Wells Fargo Charles Duncan - Piper Jaffray Maury Raycroft - Jefferies James Birchenough - Wells Fargo.

Good afternoon and welcome to the Sangamo Therapeutics Teleconference to discuss Third Quarter 2017 Financial Results. This call is being recorded. I will now pass you over to the coordinator of this event, McDavid Stilwell, Vice President of Corporate Communications and Investor Relations..

Good afternoon, and thank you for joining Sangamo’s management team on our conference call to discuss the company’s third quarter 2017 financial results. As we begin, I’d like to point out that today we will be referring to a slide presentation.

You may find a link to the slide presentation on our website, www.sangamo.com, on the Events and Presentations page of the Investors and Media section of the site.

I’d also like to remind everyone that the projections and forward-looking statements that we discuss during this conference call are based upon the information that we currently have available.

This information will likely change over time by discussing our current perception of the market and the future performance of Sangamo with you today, we are not undertaking an obligation to provide updates in the future.

Actual results may differ substantially from what we discuss today and no one should assume at a later date that our comments from today are still valid.

We alert you to be aware of the risks that are detailed in documents that the company files with the Securities and Exchange Commission, specifically our Annual Report on Form 10-K and on our Quarterly Reports on Form 10-Q.

These documents include important factors that could cause the actual results of the company’s operations to differ materially from those contained in our projections or forward-looking statements.

With me today on this call are several members of Sangamo’s senior management, including Sandy Macrae, Chief Executive Officer; Kathy Yi, Chief Financial Officer; Ed Conner, Chief Medical Officer; Michael Holmes, Vice President of Research; and Curt Herberts, Chief Business Officer. And again, we will refer to a slide presentation during this call.

Those slides are to be found on the Events and Presentations page of the Investors and Media section of our site. And now, I’d like to turn the call over to Sandy..

Thank you, McDavid, and welcome everyone to our conference call to review business and financial highlights from the third quarter 2017.

We’re pleased to share the progress from our clinical and preclinical programs with you all, and take sometime to discuss the continued growth and evolution of Sangamo, including plans for our new headquarters in Brisbane, California in the heart of the Bay Area biotech community, South of San Francisco.

And the steps we’re taking to develop the manufacturing and product development capabilities, we will need as we advance through clinical trials towards commercialization.

We’ve made great progress revitalizing Sangamo, improving our core technologies, establishing and renewing partnerships, strengthening the balance sheet and moving programs into the clinic.

In the last few months, we treated a second patient in the SB-525 program, and in our other clinical programs, patients are advancing through the screening process. Our Board of Directors has now encouraged us to be bold in planning for the company’s successful future.

The new headquarters in Brisbane, which we expect to open beginning late 2018, will provide more than just square footage. It will importantly provided us a more competitive location and workspace as we recruit the managerial, clinical, operational and manufacturing talent, we need to achieve our long-term goals.

Our roots are in Point Richmond and we’re talking to our scientists in the East Bay. We will retain our presence here and update the facility to be a research center for Sangamo. Today, we also announced improvements to our manufacturing and product development capabilities.

We have a new Head of our Technical Operations Group delighted to welcome Andy Ramelmeier, who comes to Sangamo with over 25 years of experience in pharmaceutical manufacturing, process development and technical operations at Merck, J&J, BioMarin, and most recently served a Senior Vice President, Biological Technical Operations at Portola Pharmaceuticals.

And his manufacturing experience and leadership will be extremely valuable, as we continue to develop the capabilities and resources to control quality, timelines and cost of our manufacturing and product development. I’m very happy to welcome him to the team.

As Curt will discuss later in the call, we have contracted with Brammer Bio to establish dedicated manufacturing capacity for our therapeutic programs.

Brammer has been our CMO for over 10 years, and is a leading clinical development and manufacturing organization, providing clinical and commercial supply of batches [ph] for both in vivo and ex vivo genomic therapies.

In addition, the new Brisbane headquarters will enable us to build our own state-of-the-art GMP manufacturing facility, initially focused on product development and Phase 1/2 clinical trial materials. Our goal is to establish a balanced approach for manufacturing.

We’re augmenting our current capacity with Brammer Bio and we’re complementing their expertise in clinical and commercial scale vector production with our own in-house manufacturing facility to address ongoing business development discussions and to ensure the flexibility needed to meet our growing demand for manufacturing.

This approach will allow us to take on a greater ownership of our product development process, as we continue to expand our pipeline with a focus towards the future. In addition to the lease of our new headquarters, slightly it highlights other recent advancement activities will be focused discussing on the call today.

At the start of October, we announced that the FDA accepted our IND application for ST-400, Sangamo’s gene-edited cell therapy candidate for beta-thalassemia, being developed as part of a collaboration with Bioverativ.

We believe the precision, efficiency and specificity of our zinc finger nuclease [ph] approaches significant advantages over other genome editing approaches and a randomly integrating lentiviral-based gene therapies in development. In a moment, Ed will provide you up an update on the progress of our clinical trials.

Mike will then review the recent advances and our T-cell editing capabilities and novel delivery research. Curt will outline Sangamo’s long-term operational target in new headquarters, and Kathy will summarize our financial results for the third quarter.

I’ll now pass the call over to Ed Conner, our Chief Medical Officer for an update on our clinical programs..

Thanks. Thank you, Sandy, and good afternoon, everyone, on the call. I’m very excited to share the progress we’ve made in the third quarter on our now five clinical programs. As expected, our activation of sites, scientific outreach and patients awareness campaigns are yielding results as we head into the end of the year.

I’ll go through each development programs starting with SB-525 for the treatment of severe hemophilia A. For the SB-525 Phase 1/2 study, we continued to execute and have dosed the first two patients with several more in screening to allow us to fill open slots as they become available.

Today, we’ve activated six sites and plan on opening three more by the end of the year. Our definition of success on the study is to produce sufficient levels of factor resulting in no spontaneous bleeds and no need of replacement factor products. We continue to be on track to have data from this program in the first-half of 2018.

Enrolment for SBF-9 for the treatment of severe hemophilia B continues to be a challenge, but we’re actively screening patients and anticipating activating one additional site by year-end.

For the MPS I, SB-318 and MPS II, SB-913 programs we are working closely with our principal investigators and academic centers around initiation and patient enrollment for these studies.

We currency have four sites activates on SB-318, working three more by the end of the year and five sites activated on SB-913 with two more opening by the end of the year. We are advancing several patients through this winning process. And I’m confident, we will treat our first patient very soon.

We also have greater visibility in the formation of a pipeline of patients for these studies. An important new development across the MPS programs is the movement toward enrollment of pediatric patient.

Our development strategy is to include adolescent and children in our current Phase 1/2 studies after appropriate safety information is collected in adults. In recent discussion to scientific advice with the European Health Authorities, they encouraged this approach and we plan to file a CTA to open a pediatric study in Europe next year.

I’m very excited about this, because the greatest potential benefit for our gene-editing programs that integrate permanently into the genome is in the treatment of children, to treat them before they suffer permanent irreversible disability and to provide a lifelong benefit.

Success in the Phase 1/2 study is for SB-318 and SB-913 for MPS I and MPS II, respectively, means maintaining a lowering ear and GAG levels to within a normal range while patients are off enzyme replacement therapy. Replacement of sufficient enzyme level is what drives lowering of GAG.

A number of you have asked me, what percentage of normal enzyme activity will lead to a clinically meaningful benefit? We believe consistent enzyme activity levels is about only 2% of normal, maybe the key threshold where patients could withdraw from ERT and our studies to evaluate our therapies effects on urine GAGs.

Current standard of care ERTs have never been to achieve consistent enzyme activity levels of both 2% and truthfully as no one has tested and approached like ours, they could lead to study levels above this, it is still unknown exactly what this would mean for a patient.

Our last and most recent program is ST-400, an autologous gene edited stem cell treatment for beta-thalassemia. We received IND clearance last month from the FDA. The IND was successful for three reasons. First, we have taken the time to develop the most precise, efficient and specific gene-edited product.

Second, we have a wealth of experience with the FDA and demonstrated a robust preclinical characterization of our product, manufacturing and toxicology. Third. our close collaboration with Bioverativ leverage years of experience in technical expertise between both organizations to ensure a successful submission.

As shown on Slide 15 through 17, ST-400, is a highly differentiated therapy that harnesses a natural protective mechanism of the body.

In contrast to randomly integrating lentiviral-based gene therapies in development, our approach involves non-viral mRNA delivery of the offense, a strategic advantage that allows for more controlled and precise genome editing with a potentially superior long-term safety profile.

We believe this therapeutic strategy has the potential to be a permanent lifelong treatment for beta-thalassemia with a single administration. Turning to Slide 18, we expect to have phase initiated into enroll our first patient in the first-half of next year.

With Bioverativ, we’re also continuing to work to advance the sickle cell disease program in 2018. I’ll now turn the call over to Michael Holmes our Head of Research.

Mike?.

precision, efficiency and specificity shown on Slide 20. Everyone involved in therapeutic gene editing must contribute these three attributes together when apply to clinical field. Precision is the ability to target a specific clinical development nucleotide or sequence down to a single-based therapy.

Efficiency with level of modification achieved at the desired target site. Specificity the term used to described the degree of authorized [ph] editing and must be considered in the context of precision and efficiency.

With some other hypodermic gene editing tools pushing the system to increase specificity can come at the expense of precision and efficiency. I encourage everyone’s interest in therapeutic genome editing to continue to ask Sangamo and others in the field about all three.

Turning to Slide 21, you’ll find one recent relevant example, where Sangamo has already demonstrated the precision, efficiency and specificity of our zinc finger nucleases in the application of our gene-edited cell therapy programs for beta-thalassemia and sickle cell disease.

And even more recent example comes from our T-cell oncology program to produce allogenic or universal cell therapies.

Turning to Slide 22, manufacturing the allogenic CAR-T cell therapy not only requires the integration of our CAR gene construct into the cell genome, but also the double knock-out of the endogenous TCR and B2M genes to eliminate [indiscernible] and the host immune response.

Thanks for the hard work of Gary Lee and his team, we were widely achieving very high levels of editing efficiency. Earlier this year Gary presented data demonstrating greater than 90% double knock-out of TCR and B2M and greater than 90% target integration of a gene into the TRAC locus shown on Slide 23.

More recently Gary has begun to widely achieve greater than even 99% on inefficiency with no detectable off-period activity and analyzed with the gold standard highly sensitive oligo capture assay. This is important for the production of an allogenic cell product, because of a compounding effect of multiple gene modifications.

As you can see from Slide 24, if the editing efficiency of each step is not well above 90%, the number of cells contained in all three gene modification significantly decreases.

Even at levels of 50% gene editing efficiency, the compounding effect results in only 13% of the T-cells actually having all three modification that are required generating T-cell.

This inefficiency can result in more expensive manufacturing process and runs the risk of not producing enough modified cells making it more difficult to select for and expand the therapeutic cell population.

That is why we believe the improvement to our ZFN genome editing platform is well into 10 years of operational and clinical expertise we’ve attained throughout our legacy HIV program. The increased efficiency of Sangamo to drive the development of oncologist and allogenic best-in-class T-cell therapies for immuno-oncology.

Moving on now to our LNP delivery platform, I’d like to share our latest advancement, which represented a couple of weeks ago at the ESGCT 2017 Annual Congress.

Turning to Slide 26, Sangamo scientist Anthony Conway presented new data demonstrating that we can achieve 90% protein knock-down with close to 100% gene efficiency in hepatocytes of mice with no elevation in liver toxicity at LNP doses as low as 0.20 mg/kg.

This is highly encouraging as it demonstrates extremely efficient genome editing in mice at over a long load dose than the other company approaches. Based on these data, we believe Sangamo is well within the therapeutically relevant dosing window with our non-viral LNP delivery platform.

We’re now moving forward and have already initiated series of non-human primary studies to test the efficiency and potency of our ZFN non-viral LNP delivery in larger amount in species.

Shifting to our tauopathies program, I’d like to take a moment to share some of our recent work on the development of our proprietary Sangamo AV Vector designed to more effectively cross the blood brain barrier.

As seen by the brown thing in the images on the right of Slide 27, our proprietary Vector demonstrates superior neuronal transduction in three target regions of mouse range in comparison to AAV9, in AV0 type no transit [ph] results.

In addition Slide 28 demonstrates that a single administration of our tau targeting zinc finger protein transcription factors using Sangamo’s new proprietary AV Vector results in up to 70% reduction of tau protein in the Motor Cortex, Hippocampus and Brianstem for mice.

Sangamo scientists and our collaborators for Massachusetts General Hospital will be presenting data from this program next week at the 2017 Annual Meeting of the Society of Neuroscience. And I look forward to providing further update after the meeting. I’ll now turn the call over to Curt Herberts, our Chief Business Officer for a business update.

Curt?.

Thanks, Mike, and good afternoon, everyone. Building on the progress and our clinical programs and technology innovation, as Chief Business Officer, I’m excited to discuss Sangamo’s long-term corporate strategy and how we think about business operations and planning.

As we’ve discussed before, we have a very strong platform and product pipeline, and our core business strategy is to retain programs that are right-size for Sangamo and to externalize others. For those that we retain, we look forward to integrate the business into a sustainable fully integrated biopharmaceutical company.

This strategy requires the appropriate location, people, infrastructure and capabilities to recruit too talent as we progress from an early-stage clinical company to a pre-commercial organization and then into full-fledged commercial capability.

As Sandy mentioned earlier on the call securing our new Bay Area headquarters in the heart of South San Francisco Biotech cluster is an important first step towards the realization of this updated learning [ph]. We expect these new facilities to open in late 2018, with capacity to accommodate a growing number of employees.

Our new headquarter will be strategically positioned to attract top talent across many functional areas, including business, clinical and technical operations.

Once our Brisbane San Francisco facility is up and running, Sangamo will transition to a dual play operational model with our current offices and laboratories in Point Richmond to remain as the company’s innovative research center. Today, we also announced some improvements in our manufacturing capabilities and capacity.

So it will help us better control our portfolio strategy to improve quality, timelines and costs, as we continue to advance our pipeline through clinical development and into commercialization. There are three parts to this improvement.

One, as Sandy highlighted, we acquired a talented and experienced leader, Andy Ramelmeier, who is our new Senior Vice President and Head of Technical Operations and Manufacturing.

Two, we’ve expanded our business relationship with Brammer Bio, a leading contract manufacturing organization, focused on cell and gene therapy product with particular focus on large-scale GMP, AAV manufacturing. It is important to note that, we have been working with Brammer for the past 10 years.

Our new agreement provides Sangamo the dedicated GMP capacity, as we advance our genomic products over the next several years.

Three, we are planning to build our own state-of-the-art GMP facility, which will enable us to take greater control over the entire technical operation process and to manage large-scale AAVs to support our pipeline programs and portfolio strategy. This facility will be housed in the ground floor as our new headquarters in Brisbane.

The corporate decision is to build an in-house GMP manufacturing facility with a key component in planning for the evolution of the company from a strategic business operations perspective.

Over the past 12 months we have made tremendous strides in restructuring the company, focusing the pipelines, establishing key partnership with companies such as Pfizer on SB-525 and hemophilia A and strengthening the balance sheet.

Now, with this investment in manufacturing and technical operations, we are laying the foundation for the future strategy of Sangamo, to take greater ownership in our product and to forward integrate in area where we believe we have a true competitive advantage based on our best-in-class genome editing and gene therapy pipeline.

Now we are establishing the necessary infrastructure for growth and a smooth transition when Sangamo is ready to support, integrate to commercialize our own therapeutic products. I look forward to providing further updates on our growth and expansion plans as we make progress for the opening of this new headquarters.

Looking ahead to new business development, we are evaluating opportunities to combine our T-cell gene editing capabilities with the right oncology partner who are committed to the application of genome editing for the future vision of adopted cell therapy.

We are also planning to partner our best-in-call gene regulation technology for several CNS indications. For the trial program we are currently initiating non-human primate study and I look forward to providing updates on future calls.

We believe strongly in this program and the additional investment in HP studies will maximize the value of this asset. I’ll now turn the call over to our CFO, Kathy Yi, for our third quarter financial update..

Thank you Curt and good afternoon everyone. We issued a press release earlier today that included detailed financial results for the third quarter to 2017, which are summarized on Slide 32. Total net loss for the third quarter of 2017 was $12.4 million or $0.15 per share compared with $19 million or $0.27 per share for the same period in 2016.

We ended the current quarter with $253.5 million in cash, cash equivalents and investment.

Based on our current cash position and excluding any potential new business collaborations or milestone payments, we believe we have at least 24 months of capital to advance our full lead clinical asset towards pivotal studies, transition our middle pipeline programs into the clinic and continue to optimize our technology and novel delivery platforms.

More importantly, our strong cash position includes build out of our Phase I/II manufacturing capability and facility in Brisbane, expected to be completed by late 2018. With this investment and recently secured manufacturing capacity at our CMO, we believe we are in a better position to ensure future clinical supply for our therapeutic pipeline.

As a final comment, we are maintaining our prior financial guidance and expect to end 2017 with at least $220 million in cash. We are in a solid financial position to fund our product pipeline and build out the necessary operational infrastructure for the new Brisbane facility. This concludes my remarks for today. I’ll now turn the call over Sandy..

Thank you Kathy. So in closing, I’m pleased to report that we’re continuing to make progress in the goals we laid out at the beginning of 2017. Clinical sites are activated in our lead clinical trials and enrollment is commencing with data expected in the first half of 2018.

Our optimized zinc finger nuclease technology is setting the standards for the TRAC of genome editing across the critical dimensions of precision, efficiency and specificity. We are making headway in the development of novel forms of delivery of genomic therapies, including new AAVs and LNPs.

Our collaborations in hemoglobin occupation, hemophilia A are successfully advancing these programs and we expect to continue to forge new partnerships for selected program in the future.

With this progress along with our newly announced manufacturing plants and headquarters, we believe we are laying a strong foundation for the future of Sangamo and look forward to accelerate our progress into 2018. Operator, we are now ready for questions..

[Operator Instructions] And our first question comes from the line Jim Birchenough with Wells Fargo. Your line is open..

Hi guys, thanks for taking the questions and congratulations on all the progress. So, a couple of questions, the detail around the transfection efficiency for the beta-thalassemia program is really helpful.

I’ve asked before, but just wondering if you could maybe go through in similar terms how you think about the transfection efficiency for the gene editing programs that are in the clinic right now for MPS 1 and 2 and hemophilia B and then I’ve got a follow-up?.

Okay, thanks Jim.

Mike, do you want to take that one on?.

Certainly, so I think as you’ve sort of brought up in terms of the transfection efficiency when it comes to IV PRPs, we are delivering three different vectors where each of two vectors deliver to DFNs and then the third vector delivers to donor [ph].

And in our pre-clinical studies we evaluated two things, number one, what was the appropriate or optimal dose of each vector component, as well as what was the appropriate ratio to maximize the delivery of all three components, both the DFNs as well as the donor that would allow us to achieve efficient levels of target integration into the TRAC locus and drive therapeutic levels of gene expression.

And so that’s at least from the pre-clinical work how we sort of came about with trying to optimize transfection efficiency and ensure that we were able to obtain at least a very high efficient levels of target integration to the TRAC locus..

Hey Jim, it’s Curt, just to add a couple of comments, and obviously the ex vivo 12 therapy setting is a very different situation than in vivo genome editing. So as Mike was alluding to for the [indiscernible] strategy, we think we only need 1% target integration in order to drive good therapeutic levels of protein expression.

In the ex vivo setting you are seeing for both our beta-thalassemia as well as the T-cell work we have done, as we are regularly achieving greater than 80% now 90% in terms of ex vivo gene modification, gene knockdown and we are very excited about the improvement in the technology, as well as the optimization of the manufacturing process and that really laid the foundation for the beta-thal IND that was submitted earlier this year..

And then just on the ….

I’m sorry, go ahead..

You have a follow-up question Jim?.

Yes, so just in terms of the clinical execution, could you maybe discuss some of the challenges in going from screened patients to actually dosing patients and as you look at that the pipe of patient being screened in the MPS 1 and 2 trials and the hemophilia B trial, do you have some confidence that kind of patients that are being screened are actually going to be converting into dose patients?.

And I should say that like you we want to get these patients into the trial. We wants to dose them because recently we had some MPS patients visiting us here at Point Richmond and it’s clear that the patients are waiting and need this as a solution. And I’m really confident in the work of the clinical group.

Ed, do you want to talk to that?.

Yes, I mean first I think just to respond to Sandy’s comment, I’ve gotten letters and just recently received letters from patients who are really looking to enroll in the study and I think to your point, the screening activities, there are a number of them and this is a Phase I/II study so we want to evaluate the safety in the best possible way that we can, but all the patients who are screening are committed to enrolment in the study and there is broad awareness in the MPS community about these studies moving forward..

Okay, well, thanks for taking the questions and for all the detail..

Thank you..

Thank you and our next question comes from the line of Charles Duncan with Piper Jaffray. Your line is open..

Charles, good afternoon..

Hey guys, sorry for the background noise. First of all thanks for taking my questions and congratulations on the progress in the quarter. Also relative to first patient dose, congrats on that, I can’t ask you about that anymore in the 515 trial.

But I did have a couple of question, in terms of that pediatric and adolescent patients work in MPS I and II, that’s really intriguing to me, I think it really – I really like the perspective on safety.

When would you anticipate being able to announce something along those lines?.

Ed, do you want to take that one?.

Yes, sure. So as we mentioned we met with the European health authorities who were very enthusiastic about taking our therapies which primarily integrate into the genome and can provide lifelong factor or for enzymes and we realize that for all of these studies, the greatest potential benefit is in the treatment of children.

In terms of when we would announce anything, we’re actively moving forward to identifying and working with sites, but I’m not prepared at this time to give any sort of timeframe regarding when we would announce activity in Europe..

But you mentioned we have to go through a CTA process and much of this is simply tougher and by that – those timelines?.

Right..

Okay, well I’ll look forward into that update. And then perhaps a couple of question for Curt, not sure if this is appropriate for him or anyone else. Regarding the evolutionary of the company, great to see that you are taking on a different kind of headquarters that certainly will be easier to visit with you, we’re in San Francisco.

Well, I’m wondering if you consider the possible difference in price per square foot, there is a competitive advantage gained in terms of people in hiring, really justify that change in location.

Can you help us understand were there perhaps other terms with regard to local incentives or distribution or other advantages gained?.

Hey Charles thanks for the question. Yes, so obviously Point Richmond is up in the North, you stay here. We were actually quite surprised with the abilities that they’re kind of building and comfortable with the overall cost and Kathy can speak to the individual financial aspect of that..

So, Charles, we issued our 8-K today and you’ll see that our – for the length of the read, if you look at the financial commitment that is laid out there versus what’s available in that marketplace, we’re very pleased with how we negotiated this facility and the building..

Okay, that’s helpful and last question is regarding biz dev activities, very excited to see you guys making traction, making progress with regard to oncology applications of the technology and I’m wondering if you want to lay out any timelines for potential partnering either in that or certainly for the CNS activity?.

So for both of them, we continue to be very active in this area. For oncology, as I think you and I have discussed before, the world of oncology is rapidly moving world with acquisitions and launches and prices, constantly changing the environment.

And so we are continuing to be very optimistic about this and we have the best-in-class T-cell editing that a number of people are really quite interested in. For CNS, we have looked to some of the recent deal activity and appreciate the benefit of perhaps I think some more data to our package, so as we can maximize the value of the asset.

And that’s why the recent raise that we did allows us to take this forward into nonhuman primates and we feel that that will be extremely compelling data, because I’m sure you would agree that what we’ve seen in mice at the moment is very encouraging..

Yes, absolutely and may I add also in term of launches, also acquisitions in oncology making the world change quickly, so thanks for taking my questions Sandy..

Thank you..

Thank you. And our next question comes from the line of Maury Raycroft with Jefferies. Your line is open..

Good afternoon Maury..

Good afternoon Sandy and team.

First question, a quick one on hemophilia A, I’m just wondering if the new patient dose is at the same side as the first patient or a different side?.

Ed..

The new patient dose is at the same side..

Got it..

We want to just – Ed, just underline the number of sites we have, just to remind us all..

Yes, sure. So we have six sites. Currently, we’re activating three more by the end of the year. There has been intense interest in the physician community. So we’re – and as I mentioned, we have a number of patients who are actively screening. So as slots open open up in the study and we’re able to fill those things, yes..

And those patients are in different sties?.

Correct, yes. It’s not one center, but these are patients that are across all sites nationally..

Got it. Thank you.

And then for the greater than 90% ex vivo gene-editing efficiency that you’re seeing in T-cells and that’s what the double knockout and targeted insertion, just can you remind me if you’re using electroporation for them? And if you can provide any comments on how the efficiency could translate into time and cost savings in the production process if you can provide any specific on that?.

Mike?.

So, I’ll try to provide the specifics on the delivery. In terms of the ZFN for using electroporation to deliver messenger RNA, and then as far as the donor were using AV6 to promote the efficient insertion into the track logos..

Curt?.

Yes. So when we look at the slide in the presentation where we talk about compounded deficiencies similar to compounded interest that you multiply the deficiencies by each other.

So to my point, when we were doing a double knockout, what’s the target integration? We are looking for the dental therapeutic to have – that cells have all three modifications.

And what that really means is with our 90%-plus level of modification for all three levels, we get significant synergy and efficiency in making that product, which drives down the cost of goods significantly. But more importantly, we think, we’re going to get a more efficacious product and then greater number of products out of a single batch..

Got it. Thank you.

And last question is just on the LNP delivery data that you presented at SCCT, could you remind me what thoughts are on incorporating LNPs into your strategy are? And is it just exploratory, or are you considering advancing LNPs closer to clinic?.

Mike, why don’t you start this and then I’ll follow-up..

All right. So I think, I’ll start this by at SCCT, we demonstrated that we could achieve nearly a 100% at editing in hepatocytes in mice with significantly lower levels of messenger RNAs compared to competing technologies, where we could achieve the significant levels of modification at greater than a long low-dose of messenger RNA..

And – but this is a key part of our strategy. So we feel that genome editing is in a very good place. But the success of it will be done to successful delivery.

And that’s why we have dedicated efforts within our research group to look at both AVs and novel AVs and to drive forward the LNPs, because delivery is what will make our technology available to patients..

Got it. Very good. Okay. Thank you very much..

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

Hi. This is Alex on for Ritu. Just a couple of questions on the manufacturing facility.

Can you tell us what sort of capacity we should be expecting there? Do you think that eventually we should expect the clinical programs to transition to the in-house manufacturing product? And what will be the first product that we should expect to see first data with the in-house product?.

Kathy, can you try to answer that?.

Yes. So, Alex, we are currently in the design phase right now with the building. So we’re going to release more information as we clear out our – clean out our capacity understanding and what it can be. But the entire building is about 88,000 square feet.

So it’s a significant size building that has definitely possibility for future commercialization product..

And we’re very pleased to be able to blend the work we do with Brammer and the dedicated resources they’re giving us with this future of having our own in-house capability. And without being rigid in one dose X and the other one dose Y, I think, it gives us the flexibility.

And we’re delighted to – people – Mark and the team at Brammer have been very supportive of both, also driving ahead with Board capabilities..

Perfect. Thanks for asking the questions..

Thank you. And we have a follow-up – [Operator Instructions] Thank you. And we have a follow-up question from Jim Birchenough with Wells Fargo. Your line is open..

Hey, Jim..

Hey, guys. Hi. just wondering if you’d be willing to give a little bit more detail on the timing of the two patients with SB-525.

I’ was trying to get a sense of whether we crossed the threshold of where we would expect to see liver enzyme elevation if we’re going to see them? And what kind of efficacy expectations we would have at this current dose level? Thanks..

So, sorry, Ed, perhaps you could take this one..

Yes, sure. So we’re not going to be discussing the timing of the patient just yet. But as I’d say, we’re keeping on track with that study. I’ve been very pleased with the progress thus far in terms of getting patients in, moving them swiftly through the process and getting them enrolled.

And we’re on track for all the deliverables on that study the we set out earlier this year..

And patient safety is very important to us. So we’re constantly measuring liver function tests in these patients..

Yes..

We don’t have any preconceived ideas of when you have to look. And so we will continue to look after these patients..

Correct..

Great. Thanks for taking the call..

Thank you. Thank you. And I’m showing no further questions at this time. I would now like to turn the call back to Dr. Sandy Macrae for closing remarks..

Thank you. And we just like to thank you all for your continued support and for your listening today on your questions and wish you a very good afternoon..

Ladies and gentlemen, thank you for participating in today’s conference. This does conclude the program, and you may all disconnect. Everyone, have a great day..