McDavid Stilwel - VP of Corporate Communications and IR Sandy Macrae - President and CEO Curt Herberts - SVP and CBO Edward Conner - SVP and CMO Michael Holmes - VP, Research Kathy Yi - SVP and CFO.

Ritu Baral - Cowen & Company Maury Raycroft - Jefferies.

Good afternoon and welcome to the Sangamo Therapeutics Teleconference to discuss Second Quarter 2017 Financial Results. This call is being recorded. I will now pass you over to the coordinator of this event, McDavid Stilwel, 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 second quarter 2017 financial results. As we begin, I’d like to point out that we will be referring to a slide presentation this afternoon.

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

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 our very busy second quarter. We continued to make strong progress repositioning Sangamo financially and operationally for its leadership role in the development of genomic therapies.

During the second quarter, we executed two significant transactions. We and Pfizer entered into the SB-525 collaboration for the development of the AAV gene therapy for hemophilia A. This agreement will strengthen the SB-525 which is on our program and greatly accelerate the potential global commercialization of this product candidate.

We are excited to work closely with Pfizer and with their advanced capabilities in gene therapy delivery and product developments. The financial terms were also very attractive that included a $70 million upfront payment as well as $475 million in potential milestone payments and a tiered double-digit royalty on net sales.

Beyond putting our hemophilia A gene therapy program into collaboration with the right partner, we believe the Pfizer collaboration announcement has served us an important catalyst for Sangamo raising awareness of the quality of our science amongst other potential business development partners and amongst investors too.

Six weeks after the Pfizer collaboration announcement, we announced a second major transaction for the quarter. A follow-on offering of common stock raising $78.1 million in net proceeds. The transaction allows us to broaden our shareholder base and to conclude the second quarter with a significantly strengthened balance sheet.

And Kathy will provide details later in the call of our use for these funds. Both these actions are positioning Sangamo for an exciting future, as Sangamo purchased over a year now and I am more confident than ever in the prospects for the company.

I believe the scientific platform here is unrivaled and as Curt will discuss is increasingly recognized as such by potential external partners, we are looking to our future with editing a revolutionary used therapeutic technology. In scientific presentations this year, we have been demonstrating recent advancements in our zinc fingers.

We believe zinc finger nucleases are the most flexible, advanced, precise and specific editing technology unrestricted by guide sequences and relatively engineered by Sangamo scientists to eliminate off-target activity. CFMs are capable of knocking out or delivering new genes to any location in the genome.

Our initial applications for this technology are in vivo genome editing where we insert a new gene into safe location within the genome deliver. This approach is more advanced in gene therapy with this therapeutic gene being integrated into the patient’s DNA allowing for the gene’s retention as cells to fight.

In a moment, Ed Conner will provide an update on our four clinical trials including the first ever in vivo genome editing studies. We believe that the future applications will be more exciting still.

The investment to deliver technology, not just at Sangamo, but by many public institutions and private enterprises is searching through available technologies we are currently able to deliver genomic therapies to the liver, brain and eye. I believed before long the problem of delivery will be solved.

Scientists and Sangamo and elsewhere will understand how to put genomic therapies into other tissues, the skin, muscle, heart and lungs. This delivery will open the field to such exciting possibilities of being able to truly cure disease and Sangamo will be very well positioned with the technology for editing anywhere in the body.

Sangamo’s zinc finger protein based approaches to gene editing; gene regulation and cell therapy are therefore well poised for this future. In the mean time, we are focused on creating value with our research engine and Michael Holmes will highlight progress in central programs later in the call.

Our platform creates more opportunities that we are able to take forward ourselves. To maximize the value of this platform and to spread the use of the technology, we believe that externalization of R&D must be a central part of our strategy. In addition to the new Pfizer collaboration, we’ve already partnered to hemoglobinopathies with Bioverativ.

The IND for beta-thalassemia is being completed as we speak and we will press release once it is being cleared by the FDA. We expect in 2018 to advance this program into clinical trials. We are looking to partner additional improvements, especially from a special expertise or franchise is required to maximize the value of the asset.

We are evaluating opportunities to pair our T-cell editing capabilities with the right oncology partner. Ideally, one with a strong library of targeting for immuno oncology applications.

We also expect eventually to enter partnerships to advance our gene regulation technology for CNS indications including ZFP mediated tau lowering for Alzheimer’s disease and other Tauopathies.

We are also receiving inbound interest from many other companies who are beginning to evaluated genome editing as a novel solution for other diseases in different tissue types and therapeutic areas. This company feels very different from when I started a year ago.

The various parts of Sangamo, research and development, manufacturing, business development are coming together to advance our programs through internal efforts and externalization.

We have a new direction, a stronger sense of purpose and as the ecosystem of the genomic technologies continues to develop and deliveries challenges are solved enabling gene editing in tissues throughout the body, Sangamo is well positioned to lead the field developing revolutionary immune medicines. It truly is an exciting time for the company.

I’ll now turn to Kathy Yi for a financial update.

Kathy?.

Thank you, Sandy and good afternoon to everyone joining on the call. We issued a press release earlier today that included detailed financial results for the second quarter of 2017, and updated guidance for the remainder of the year, which I will summarize on Slide 11.

Later, I’ll be happy to answer questions about the quarterly financial results that are extensively laid out in the press release. In my comments today, I’ll focus on the cash utilization portion of our updated guidance. This updated 2017 guidance reflects $70 million of upfront cash received from Pfizer during the second quarter.

We are accounting for this payment as deferred revenue and recognizing it on a straight-line basis over a 32 months period. Including funds raised in the follow-on offering we completed in June, we ended the second quarter with $266.5 million in cash, cash equivalents and investments.

Based on our quarter end cash position, and with projected full year operating expenses revised partly lower to a range of $90 million to $100 million we expect to end 2017 with at least $220 million excluding any potential new business collaboration or milestone payments.

We believe we are in a solid financial position to fund our lead clinical development and middle pipeline programs through a key value inflection point.

In the months ahead, we anticipate proof-of-concept data from our four lead clinical trials, the manufacture of pivotal clinical trial materials which we recently initiated, the progression of our Fabry gene therapy program, and Bioverativ partnered beta beta-thalassemia and sickle cell disease programs into the first in human studies, as well as ongoing investments in our core zinc finger approaching technology platform and novel delivery modalities.

We believe Sangamo is in solid position to expand our future pipeline and unlock value for our shareholders. And with that, I will turn the call over to our CMO, Ed Conner for an update on our clinical programs.

Ed?.

Thanks, Kathy. I am very pleased to share a status report for activities across our four clinical programs. I’ll start with SB-525 for hemophilia A on Slide 14. We opened our first two sites in the second quarter that have now two patients who are qualified to enroll based on serology testing.

We expect to do as the first patient in the study this month as we coordinate the 24 hour observation visit at the participating site. We also have a number of additional sites being activated this month and next and expect to have six study sites opened by mid-September.

One common question I am asked is, how do evaluate success for the hemophilia program and it really comes down to the factor activity levels. For hemophilia, these activity levels correlate tightly with the outcomes, patients and physicians care most about namely reducing spontaneous bleeding and use a recombinant factor replacement therapy.

Data show that once factor activity levels exceed 12% incidence of spontaneous bleed drops to near zero and patients don’t need factor replacement process. Success for this study then is having factor activity to be well above that level while avoiding very high levels exceeding normal as there maybe the potential for clotting events.

Turning to Slide 15, I’ll move on to SB-FIX for hemophilia B. Enrollment in this study remains challenging as we are behind other programs with promising data. We currently have four sites opened and expect to open an additional site in September.

Patients and physicians continue to be interested in this program as it has potential as the treatment for children to provide life-long production of factor levels that eliminate the risk of bleeding and use of replacement products.

On the next slide, starting with SB-318 for MPS I, we have two sites opened currently and expect to have an additional six sites, eight sites in total open by the end of September. We have patients in screening and more scheduled to screen next month.

Regarding SB- 913 for MPS II, we have one site open and expect to have all seven sites opened by the end of September. We continue to screen patients and hope to have a qualified patient identified soon for this program.

I’d like to take a moment now to discuss what success looks like for the MPS program, because it is a bit different than for the hemophilia programs. For the MPS programs, success of having patients with stable or reduced GAL levels while off of ERT.

To walk through this a bit, I want to briefly review MPS biology which starts with enzymes that are deficient in MPS I and MPS II. These enzymes act within the lysosome. The lysosome is an organelle within the cell and this means, if these enzymes are present in the blood stream, they have to enter the cell and they have to enter the lysosome.

That’s true for enzyme replacement therapy and it’s true for our gene editing program. A point with that, because while we can sample the blood to look for enzyme levels there, what we really want to know is what is going on without the lysosomes. Specifically, it is the wave product of Glycosaminoglycans or GAG being reduced.

Fortunately, we can measure GAG in the urine and this gives us an estimate on what is going on in the cell. Success for the MPS programs in the Phase 1/2 studies is to first see the enzyme levels are being produced by the liver and circulating in the blood. We can do this even in patients who are taking ERT to get – ERT as short.

Serum levels and the enzymes are back down to their very low levels within hours of the infusion. So if we take a trough for example or a sample drawn prior to the next dose of ERT, we will be able to evaluate what therapy is producing more enzymes in the liver and circulating in the blood.

Second, we can value the GAG levels in the urine to see if they are stable or dropping further to patients on ERT. Importantly, if these enzyme levels are elevated in the blood and GAG levels in the urine are stable or declining, we will give patients the option of withdrawing ERT to see if GAG levels continue to stay well.

So success then in these studies will see patients being able to have stable enzyme levels and low GAG levels while off of ERT. Clinical outcomes are of course the most important; given that we are starting these outpatients and maybe difficult to show large changes that they have linked with the disease for years.

The integrated benefit we need to treat children and we are engaging with health authorities by the end of the year to determine the appropriate task to do that. In closing my section, I want to mention that we were just at the MPS patient meeting last week and there was a great deal of interest.

We have patients in screen and in rare disease trials having faced up this most important because it gives patients the ability to go to sites they know and see physicians they know and trust.

Knowing will have a strong roster of our sites up and running by the end of September with good geographic diversity and very strong investigators at top treatment centers gives me great confidence in our ability to enroll these studies. I’ll now turn the call to Michael Holmes, the VP of Research for an update on our preclinical pipeline.

Mike?.

Thanks, Ed. As Sandy mentioned earlier, Sangamo is evolving rapidly as a company that has tightly integrated and focused on achieving common goals. Research is working closely with our internal and external partners on clinical and product development efforts.

While our 20 year history has been discovering ground-breaking science, our future is taking our innovative discoveries for the development of new human therapeutics. Today, I’ll provide a brief update on our middle pipeline including our Bioverativ partnered product candidates for beta-thalassemia and sickle cell disease.

Our AVG therapy for Fabry disease and our ZFN media in CAR T and TCR immuno therapies for oncology. Turning to Slide 19, I’ll start off with our Bioverativ partnered cell therapy program for beta-thalassemia and sickle cell disease.

Both diseases are inherited, genetic blood disorders caused by two different mutations in the beta-globin gene, which we can address with in common approach using our ZFN-based cell therapy platform.

Our therapeutic product candidate, ST-400 and BIVV-003 for beta-thalassemia and sickle cell disease respectively are designed as the target cell therapies made from a patient’s own hematopoietic stem cells.

As shown in the cartoon on the bottom right of the slide, these stem cells are identified from the patients and genetically modified ex vivo using ZFN can increase the expression of Gamma or field globin gene while reducing expression of the mutated beta-globin gene.

This results in the production of normal function fetal hemoglobin when the modified stem cells differentiate into red blood cells inside the body. This is a highly differentiated therapeutic strategy that harnesses a natural, protected mechanism of the body.

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

We believe this product strategy has the potential to be a life-long treatment for both beta-thalassemia or sickle cell disease with a single administration. Our IND application for ST-400 is being completed as we speak and we will press release once the IND is cleared by FDA. We plan to initiate the Phase 1 2 trial for ST-400 in 2018.

We are also working with Bioverativ to complete preclinical work on for the IND application for our product candidate for sickle cell disease. We expect that IND to be filed in 2018. Moving on to ST-920, our gene therapy candidate for Fabry disease on Slide 20. Fabry is a genetic, X-linked lysosomal storage disorder caused by mutation in the GLA gene.

This mutation resulted in missing or low levels of alpha-Gal A enzyme and the buildup of toxic lipid molecules in cells leading to a range of symptoms and life threatening complications. Current treatment options for patients are very limited with only one enzyme replacement therapy approved in U.S.

and there remains significant unmet need for LS burns and more clinically effective therapies.

In contrast to ERT, which patients must take for the rest of their lives, ST-920 is an AAV-cDNA gene therapy designed as a one-time long-lasting treatment with the potential to provide durable continuous production of the alpha-Gal A enzyme at therapeutic levels.

Given most thesis of Fabry are diagnosed, we believe the conventional gene therapy is a suitable therapeutic strategy for the Fabry patient population. Based on data from preclinical studies, we expect ST-920 to have several advantages over ERT including greater clinical efficacy and better safety profile.

Preclinical IND enabling work for ST-920 is expected to be completed in next year for expected IND filing in 2018. Finally, there has been a lot of excitement in the oncology field recently as the FDA set PDUFA dates for two daily applications CAR-T therapies in the coming months.

These are very same milestones for the field and most importantly for patients in desperate need of new transformative therapeutic options.

Turning to Slide 21, I would like to highlight the technical advantages of Sangamo’s ZFN in immuno oncology and the recent advancements we have made towards the development of best-in-class CAR-T and TCR-based therapies.

We believe this next wave of immuno therapies will have even therapeutic profile by harnessing the precision and efficiency of our optimized ZFNs.

Our most recent data on T-cell modification shown on the right side of the slide, demonstrates the pure gene editing efficiency with greater than 90% modification for both knock out of HLA and endogenous TCR genes and target integration of the CAR trans gene.

The higher cell modification efficiency and simultaneous knock out of multiple endogenous genes via genome editing not only significantly reduces manufacturing costs but also provides several advantages over randomly integrating retroviral based approaches including generation of T-cells that exhibit more consistent and better regulated TCR CAR expression and activity, improved anti-tumor activity and durability and a superior safety profile.

Furthermore, our ability to specifically knock out HLA endogenous TCR genes allows us to pursue an allogenic cell therapy strategy to create the universal or so-called off the shelf immuno therapy for all patients in comparison to an autologous cell therapy strategy manufactured on a per-patient basis.

We and others are very excited about the therapeutic potential of our oncology program and the preclinical programs I just mentioned and I look forward to providing future updates on the progress towards the clinic. Thank you. And I’ll now turn the call over to Curt Herberts.

Curt?.

Thank you, Mike. As we developed our technology over the past several years, we focused on building out platform – the next ZFN media to genome editing, highly differentiated from the competition and to create best-in-class human therapeutics. From our perspective, there are three main criteria to achieve this goal.

One, precision, the ability to target any desired nucleotide in the genome, two, efficiency, the ability to create a permanent targeted double – with a specific nucleotide center and three, specificity, the ability to cut at the targeted nucleotide of interest without cutting elsewhere in the genome.

These are the three attributes that we believe are required for editing technologies that will ultimately become therapeutic products for patients, products on which we will build our long-term business. Over the years, we have assembled the library of thousands of individual well characterized zinc finger approaching.

More recently, we have advanced a series of new ZFN architecture which has greatly increased the flexibility of the platform. This year, Michael Holmes and Ed from our research team have been presenting on the new ZFN architecture and other platform improvements at leading scientific meetings.

We now have a ZFN platform with an unparalleled design density with thousands of one and two finger modules in our library and an array of links attaching the zinc fingers and the top-line nucleases. We are able to assemble many highly specific ZFN pairs for any chosen target sites. So let’s discuss why this mattered practically.

Turning to Slide 24, with the origin of DNA around the beta globin genes, for any given 20 base per window we have on average 450 distinct ZFNs can figure right that choose from out of an existing library. Across the 400 base target sites pictured here, we have thousands of distinct ZFN options to start developing that clinical leads.

Other editing technologies require an RNA guide have very few options. Only one or two potential clear sites have 20 base starts to DNA but significantly limits their ability to design an optimal clinical grade reagent for desired targets. Turning to the next slide, so why does very high design density matters.

Working in the therapeutic genome editing field has its challenges. Various criteria can make developing a clinical lead difficult refractory chromatin can limit access to the chosen site, reasons of homologue and other genes and pseudogenes may cause binding and – in unwanted locations.

These real world restrictions limit the ability of many less flexible genome editing technologies. Most importantly, the very high design density of our ZFN library has real world operational advantages and allowing us to choose a final ZFN pair with an optimal profile.

That is to say high on-target activity and minimal to know off-target cutting to allow us to take the best product forward into clinical development and eventually commercialization.

With such a robust library of well-characterized zinc fingers, we can now quickly develop a set of lead clinical grade ZFN that can be taken into the next step of full characterization and specificity assessment. Slide 26 shows an example of the progress that proves that point.

Last year we were challenged with CEP 290 for LCA 10, a severe retinal dystrophy as a knockout target. Ten days later, we had lead candidate to precisely and efficiently target the single mutation that causes LCA 10. No other editing technology can do this to spec with that level of quality and speed.

We have similar examples where we can precisely target validated genes such as the sickle and beta globin, also on a anti-trexin and DCMA just to name a few clinically relevant targets. Finally, on Slide 27, you will find a representation of our technical abilities on the [Indiscernible].

This is in partnership with Bioverativ with a focus on autologous genome edited stem cells where we were able to target a specific store-based therapeutic point for the genome with a very high level of on-target modification in the context of minimal off-target – which is at or below the level of detection by the most advanced state-of-the-art assay from the field.

Importantly, we are able to accomplish this result at clinical scale and in currently relevant cell sites which many other groups again could demonstrate. For genome editing to yield a best-in-class therapeutic product we believe this is the type of profile that is going to be required.

As Chief Business Officer at Sangamo, I am responsible for developing a comprehensive business strategy that ties the fundamentals of the technology to what we can do for patients.

I am extremely excited to have a platform of so-called opportunity in trying significant value inflection points across many different therapeutic areas, and product strategy. Potential collaborators are taking note of these advancements that differentiates therapeutic zinc finger genome editing from other technologies.

We are also very pleased with Sangamo’s unambiguous intellectual property position for zinc finger and other clinical attributes when – goals focused on products for patients.

As other aspects of genomic medicine such as delivery continue to advance, we believe that potential applications for gene editing will expand rapidly and we are very excited to have what we believe is the best platform for therapeutic genome editing. With that, I’ll turn the call back to Sandy. .

Thank you, Curt. We are pleased to report our progress today, a continuation of the studies and strides we have been making throughout this year. We are building Sangamo into a company that has the infrastructure and management capabilities to reliably deliver on our goals.

We are solidifying the clinical operations capabilities which we need to recruit and execute our rare disease studies. For me it’s not about the enhancement of the first patients into these studies which will come soon enough, but rather the confidence that we have the machinery in place to fill the trials steadily.

That has to do with which we are building Sangamo’s development capabilities. Similarly with our middle pipeline, I am very pleased to report progress towards INDs and 2018 clinical trial starts.

Next year, these will be our newest clinical programs and with Sangamo’s robust R&D engine, we have programs that will advance from discovery into preclinical research in preparation for INDs. There is such tremendous value in Sangamo’s platform which is a potential to deliver new assets for movement through early research and into the clinic.

Curt discussed the value of the ZFN platform. Biotechnology as both science and business and we are marrying our scientific excellence with clinical delivery and commercial planning and are thinking holistically about the strategies to create the most value from our assets.

We will forward integrate to develop and commercialize certain programs ourselves, but we will also continue to externalize R&D through collaborations in order to advance assets. Our truly ventured developed and partnerships, we are very fortunate to represent with such a rich pool of opportunities. Operator, we are now ready for questions..

[Operator Instructions] Our first question comes from Ritu Baral with Cowen & Company. Your line is open..

Good afternoon guys. Thanks for taking the question.

I wanted to ask about the MPS I – actually the MPS programs in general and how you are looking at GAG levels and enzyme trough levels? What is your target enzyme trough level given what’s necessary in MPS I to – I guess, clinical benefit and you mentioned you are in MPS I, does the trial protocol gives any opportunity to measure CSF GAG levels as well? And I’ve got a follow-up..

Okay. Thank you for your question. Nice to hear your voice. I am kind of passing this on to Ed..

Sure. So, to answer your second question first, we are looking at the CSF and looking at levels of enzymes there.

For your first question, regarding the levels that are needed of the enzymes in the bloodstream, we know that in patients on ERT they are going to be as essentially their baseline levels which is as or near zero and in talking to investigators and in talking to physicians who treat this disease, they think the amount of circulating levels that we need to get our quite small to show clinical benefit.

And we need them above 2% or 5% is likely to have clinically meaningful benefit if it’s circulating continuously being produced by the liver. We are tracking these levels and the real test as I mentioned earlier will be following the GAG levels, because you want to know what’s happening in the – what’s happening in the lysosome.

So once we see these levels start to come up in the bloodstream following the urine GAGs and seeing those GAGs stay low after patients have discontinued their ERT, but to continue to benefit from our therapy will demonstrate success for this study. .

Got it. And then a quick question on the hemophilia A program. You mentioned patients qualified for enrollment, are there any gating factors for treating these patients? And also, Kathy mentioned some expenses for production of pivotal trial material.

Was that for hemophilia A or is that for another program?.

So, let me answer, give you the answer to this.

Do you want to first answer with the patients?.

Yes, so, for the patients, there is nothing gating in terms of screening activities. It’s really just a logistic issue because it requires an overnight stay. And so you need to slot in for those spots. So, that’s really what we are waiting on in terms of getting that set up in the participating site. .

Kathy?.

Yes, so, pivotal clinical trial materials is for our four lead assets mainly the MPS I and II. .

Got it. Thanks for taking the questions guys..

I want you to take away from this is, we are thinking of this more holistically, more like a – company would where you think of – you have to plan ahead for the next stage of development have the clinical development pawn in place after manufacturing done to minimize any delays between the different phases of development. .

Thank you. Our next question comes from Maury Raycroft with Jefferies. Your line is open..

Hi, thanks for taking my questions. I just had two quick ones. I was wondering for AAV 2 and six shutting or clearance over time.

If you could just remind me what the data is in non-human primates and is it’s strictly dose-related?.

Thanks for your question, Maury.

Michael, could you answer that one?.

Yes, in terms of vector shutting data, so we did monitor this in our preclinical studies including non-human primates and this was tracked over the first several weeks where we could detect showing the vector in a lot of the secretion.

But it rapidly cleared in non-human primates and we didn’t see any real differences in sort of the AV 6 vector versus other vectors that have been published on that is clinically including AV 2 as well as AV 8..

Okay, great.

And then for the sites that have been selected, so far that are posted on clinical trials, I was just wondering if there is strategic rationale for those?.

So, that’s another good question, Ed, how do you think about choosing sites?.

Well, it’s first and foremost based on sites and investigators with clinical expertise and not just in treating the disease but in doing all the parts of study enrollment making sure that the data is entered appropriately and that the data can be used in registrational activities later on.

But in other factors to consider is, because these trials are fairly involved with clinical trials. As you want to make sure you have good geographic spread within the United States. So that patients are able to hopefully go to a site where they are being treated or if not, not have to travel a great distance.

So, for me, it really both with them trying to lessen the burden as much as possible for patients enrolling in these studies, but also making sure that we have clinical trial sites that are excellent in conducting clinical trials, but also excellent in treating these diseases..

Okay, great..

Okay..

Thank you very much..

Our next question comes from Charles Duncan with Piper Jaffray. Your line is open..

Hi this is Sarah on for Charles. Good to see some of the progress on the clinic over the past couple of months.

On MPS I and II, in the slide that looks like you expect preliminary data around year end or early 2018 and can you just remind us what you include in that category in terms of number of patients and efficacy or safety parameters that you will share?.

So, that’s a good question and as we said before, we are looking for the clinical trials – each of the clinical trials to come in towards year end or most likely in early 2018 and we will report the data as we see clinically significant important results.

So it will all depend how the data comes out and how many patients are we have treated at that point..

Okay, thanks. And just one follow-up on the MPS indications. Can you speak about the rate of change in patients screened over the past couple of months and whether that’s picking up and then along those lines, how much awareness of this program do you think there is among the….

We are trying, and I will pass this over to Ed, but just to be clear, you can’t screen patients until a trial site is up and running. And so….

Yes, so, in rare disease in – rare disease really enrollment all those follows site activation. You need to get the sites opened because you can’t start treating patients until you have an open site.

And so the flurry of activity that we are seeing this month and next, we’ve already seen – to answer your question directly, we’ve already seen an increase in screening that’s been steadily growing over these last few months and as we now open the remainder of our sites, both for MPS I and MPS II, I expect to see that to continue to grow substantially..

Great. Thanks for taking my questions..

It’s our pleasure. .

Thank you. [Operator Instructions] Our next question comes from Jim Birchenough with Wells Fargo. Your line is open..

Hi, it’s Nichol for this Jim this afternoon. Just a couple of questions. For the MPS programs, is there any reason to hope that you would get better partitioning of enzyme into the CNS and what you have seen with current ERT? And I have a follow-up..

So, it’s a good question, it’s an important question, because it’s an important thing that patients and their families are asking about and we won’t be very clear and careful and the evidence that we talk about.

And so, we have seen evidence in mice and Mike, do you want to talk about evidence?.

So, in our preclinical studies in the MPS I and MPS II, mouse models, we did see that in treating these mice, that we had seen very large amounts of enzyme being produced and being secreted in the blood, and that this did seem to provide some protection with regards to the degradation that you normally see in terms of neurocognitive effects in the mice.

And we somewhat hypothesize that given that we are able to - in using the IV ERT approach in mice, because we are able to achieve a constant high level amount of enzyme that we were able to get some of this enzymes across the blood brain barrier and provide protection or at least the breakdown of GAGS that would prevent the neurocognitive degradation that you normally see in these mice.

So, I think that’s what we saw in our preclinical studies, but as Sandy mentioned, we just want to be very careful in terms of – as we sort of extrapolate what we’ve seen in mice until what we might potentially see in our clinical studies. .

And all of us in this industry have seen most mice results that have not been reflected in clinical studies. However, these are remarkably clear in the differentiation of the product provides. So it encourages us and the patients and families and this field..

Okay and can you – is it possible to determine what proportion of neuro protection, you are saying you are not ablating neurocognitive..

I think that would be, it’s tempting and we hope that we are able to provide that neurocognitive protection to the patients but the translatability of this is – I repeatedly tell people, this is cutting-edge clinical science where with the first time that any of these patients has sort of a constant supply of the enzyme until the translation between that the therapeutic effects will really be determined as the studies evolve.

.

Okay, and then, in terms of potential partnerships, T-cell-based therapy, can you give some guidance as to when one of those might be consummated?.

Curt, do you wish to answer that?.

I think what I will say is that, we are very impressed with the data that that Mike has shown both in terms of the level of single knockout, double knockout rate at the 90% and especially with the level of targeted integrations greater than 90% in that context. No other parties in the entire area are really able to achieve this.

And so, we are currently evaluating a number of options here. .

We will of course press release on it when we – if we succeed in a relationship..

Okay, thank you very much..

Thank you. I am showing no further questions in queue at this time. I’d like to turn the call back over to Mr. Macrae for closing remarks. .

Thank you. I would like to thank you all for your continued support of Sangamo and your interest in what we do and wish you a great afternoon and evening. .

Thank you. Ladies and gentlemen, that does conclude today's conference. Thank you very much for y our participation. You may all disconnect. Have a wonderful day..