SGMO Sangamo Therapeutics

McDavid Stilwell SVP, Corporate Communication and IR
Sandy Macrae CEO
Sung Lee CFO
Stephane Boissel Head, Corporate Strategy
Adrian Woolfson Head, Research and Development
Mark McClung Chief Business Officer
Bettina Cockroft Chief Medical Officer
Maury Raycroft Jefferies
Gena Wang Barclays
Whitney Ijem Guggenheim
Ritu Baral Cowen
Yanan Zhu Wells Fargo Securities
Call transcript

Good afternoon. And welcome to the Sangamo Therapeutics Teleconference to Discuss First Quarter 2020 Financial Results. This call is being recorded.

I will now pass you over to the coordinator of this event McDavid Stilwell, Senior Vice President of Corporate Communication and Investor Relations.

McDavid Stilwell

Good afternoon. And thank you for joining us today.

With me this afternoon on this call are several members of the Sangamo senior management team, including Sandy Macrae, Chief Executive Officer; Sung Lee, Chief Financial Officer; Stephane Boissel, Head of Corporate Strategy; Adrian Woolfson, Head of Research and Development; Mark McClung , Chief Business Officer and Bettina Cockroft, Chief Medical Officer.

Slides from our corporate presentation can be found at our website,, under the Investors and Media section in the Events and Presentations page.

This call includes forward-looking statements regarding Sangamo's current expectations. These statements include, but are not limited to statements relating to our pipeline of genomic medicine product candidates, our ability to develop, obtain regulatory approval 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 trials to provide clinical benefit to patients, the potential to use certain technologies to develop our therapies, the financial and operational impacts of our collaboration, plans and timelines for building and opening manufacturing facilities, the evolving COVID-19 pandemic and our expectations regarding our financial performance and resources.

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 most recent quarterly report on Form 10-Q filed today, and our annual report on Form 10-K.

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 discover non-GAAP financial measures. 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 measures discussed on this column are included in today's press release, which is available on our website.

And now, I'd like to turn the call over to Sandy.

Sandy Macrae

Thank you, McDavid. And good afternoon to everyone on the call.

Typically, when I come together with the Sangamo executive team to talk to you about our business every quarter, we are in the boardroom of the headquarters in Brisbane, California. Today, due to the COVID-19 pandemic, we're all in our home offices, one of the many adaptations we've all had to make in these unprecedented circumstances.

At Sangamo, over the last two months, we've worked together to minimize any impact of the pandemic on our business, and I believe that we are in a very strong position. Last month, we were very pleased to announce the closing of our collaboration agreement with Biogen to develop gene regulation therapies for Alzheimer's disease, Parkinson's disease, neuromuscular and other neurological diseases.

We have received from Biogen $225 million in proceeds from the sale of the stock and an additional $125 million upfront license fee payment.

With this $350 million received from Biogen and in addition to the $363 million in cash resources that we reported on our March 31st balance sheet, we believe we have the financial strength to execute on our wholly-owned and partnered development programs beyond multiple important milestones including the potential filing of the BLA for SB-525 for hemophilia A.

In response to COVID-19, Sangamo has prioritized employee safety and welfare, while responsibly advancing the business.

Following the shelter-in-place guidance from government authorities in the middle of the March, we asked all non-lab employees to work from home.

We also implemented modified lab operations and updated safety protocols to continue critical research and manufacturing work while protecting employee safety and achieving to social distance guidance.

In the labs, we are strategically prioritizing projects to keep our business on track, including focusing on research activities for partnered programs. We're also conducting clinical activities cautiously, so as not to contribute unnecessarily to the constraint in the healthcare system while staying in close communication with trial sites to protect the safety of the patients in the trial.

We continue to be optimistic with our AAP manufacturing facility and our Brisbane headquarters will be operational by the end of this year.

We also expect our cell therapy manufacturing units in Brisbane, California, and in Valbonne, France to be open by the end of 2021.

Furthermore, we’re keeping in regular contact with our CDMO partners, and as of now do not anticipate any COVID-19-related manufacturing delays with our activities.

Despite the challenges of the current environment, we continue to actively pursue new partnerships, both inbound to access new technology, as well as out-licensing deals such as the established ones that we already have with Pfizer, Gilead, Sanofi, Biogen, Takeda. An important example of an inbound partnership is our recently announced collaboration, an exclusive license with Mogrify, Cambridge UK company, developing local cell conversion technology that has the potential to serve as a renewable cell source.

Under the terms of this agreement, Sangamo aims to use Mogrify’s proprietary cell conversion technology to develop allogeneic zinc finger protein, gene engineered, CAR-Treg therapies using iPSC derived regulatory T cells. This license agreement will diversify our options, and complement our current efforts as we develop off the shelf allogeneic CAR-Treg cell therapies.

We believe that this exciting collaboration has the potential to accelerate the development and manufacturing of novel renewable cell source, Treg therapies.

We are also continuing our discussions on outlicensing opportunities.

As a reminder, our strategy for collaborations is to advance select development programs in partnership with bio pharmaceutical companies in situations where we believe that a partner's financial resources or clinical and therapeutic area expertise may enable more rapid development and the feasibility of new treatment to patients.

Before I turn the call over to the team, I want to update you on some of the recent transitions amongst my direct reports that are a natural part of Sangamo evolution as we advance our clinical development and strategic partner.

While for the last few years we’ve added Executive Vice Presidents to lead manufacturing, legal, finance and R&D, in our latest executive appointment, as we look to the future and perceive the need for commercial development expertise and capabilities, Mark McClung has joined Sangamo, as Chief Business Officer, and now leads commercial, strategic planning, alliance management and corporate and business development.

Mark has a distinguished career leading commercial organizations including GSK, Onyx and Amgen, and highly competitive therapeutic areas that require a deep scientific knowledge and include innovative products have disrupted the standards of care and where successful product development occurs as a result of a tightly integrating patient and physician insights into late stage clinical development programs.

Stephane Boissel, our EVP of Corporate Strategy will leave sign Sangamo at the end of July and plans to return on an entrepreneurial project. Stephane joined Sangamo in 2018 after we acquired TxCell, know Sangamo France, which Stephane led as CEO. His energy and vision resulted in a recent Biogen transaction, which is one of the largest preclinical cooperation deals ever in the biopharmaceutical industry. Stephane’s impactful contribution to Sangamo will endure for many years.

With that, I will turn the call over to our Chief Medical Officer, Bettina.

Bettina Cockroft

Good afternoon.

In light of the COVID-19 pandemic, Sangamo is working very closely with all our clinical trial sites partners to devise individualized plans, to protect the safety of every patient enrolled in our study, as well as the continued integrity of our trial data. After we have established a plan for each patient, we work with sites and IRBs to establish appropriate procedures.

In some cases, trial site partners have been reducing or pausing clinical trial work to redirect capacity and resources to COVID-19 patients. At other sites, clinical trial patients are still being screened and enrolled but may not be dosed until an appropriate time is identified.

In addition to adapting our clinical operations to this new situation, we are implementing procedures now, so that as the COVID-19 crisis subsides, we'll be able to resume operations as quickly and as efficiently as possible. To do this, we're keeping in close contact with our trial site partners and continuing to identify potential patients that may be suitable for enrollment.

In some cases, we're also using this time to further optimize clinical operations processes and engage with regulatory agencies. We're also working closely with our collaboration partners to track the spaces [ph] of our joint development programs.

Turning now to some clinical updates, commencing with SB-525 or PF-07055480 hemophilia A gene therapy. We transferred operations of the fully enrolled Phase 1/2 ALTA study to Pfizer along with the IND at the end of last year.

We're working closely together with Pfizer to identify an appropriate opportunity this year to provide an update on the results that were shared at ASH 2019 from the high dose expansion cohort. Pfizer continues to target, dosing the first patient in the Phase 3 study in the H2 2020.

Pfizer is working to minimize any potential disruptions to the schedule due to the ongoing COVID-19 pandemic and continues to recruit patients into the Phase 3 lead-in study. Pfizer recently updated with the Phase 3 study protocol and have informed us that they plan to provide additional updates on the Phase 3 trial in due course.

Moving now to our wholly-owned gene therapy, ST-920 for Fabry disease. We’ve successfully screened and enrolled patients into the STAAR study, and we are waiting for a safe and appropriate time to initiate dosing the first patient.

In conjunction with our partner Sanofi, we're evaluating gene-edited cell therapies for two hemoglobinopathies ST-400 for beta thalassemia and BIVV003 for sickle cell disease. ST-400 and BIVV003 are both designed to induce the synthesis of fetal hemoglobin. This was achieved by gene edited knockout of the erythroid specific enhancer the BCL11a gene, which encodes a strong repressor of the gamma globin gene. In beta thalassemia, if fetal hemoglobin is expressed at high enough levels, it may substitute for patients absent or impaired levels of beta globin.

We have enrolled and dosed five patients into the THALES study evaluating ST-400 for beta thalassemia.

In sickle cell disease, increased fetal hemoglobin synthesis may provide the patients with functional hemoglobin and help down regulate the abnormal sickle hemoglobin that results in painful sickle cell crises and other disease features.

Sanofi has also been enrolling patients into the PRECIZN-1 study evaluating BIVV003 in sickle cell disease and dosed the first patient last year.

New analysis of the study’s data will be shared when the two studies have accumulated a sufficient number of the patients and follow-up. No additional beta thalassemia patients in the THALES study will be treated until the data from both studies has been collected and analyzed. Sanofi will in the meantime continue enrolling sickle cell patients into the PRECIZN-1 study.

We will look for an appropriate time to present data from both these studies at a future date.

I'd like to conclude by addressing a few programs that we are monitoring closely with regard to potential impact by COVID-19.

We continue to make progress with additional regulatory approvals for the Phase 1/2 STEADFAST study, evaluating our first-in-human CAR-Treg cell therapy TX200 in HLA-A2 mismatched kidney transplantation.

We expect to dose first patients in this study in 2021.

Moving on now to KITE-037, an allogeneic and anti-CD19 CAR-T cell product being developed by Kite, a Gilead company. Kite has informed us that there is a potential for a COVID-related delay to the initiation of the KITE-037 clinical trial.

I will now turn the call over the Sung for an overview of the financial results. Sung?

Sung Lee

Thank you, Bettina, and good afternoon, everyone.

We're pleased to share our financial results for the first quarter of 2020. We recorded a net loss of $42.9 million or $0.37 per share, compared to a net loss of $42.2 million or $0.41 per share for the same period in 2019. The revenues were $13.1 million, compared to $8.1 million for the same period in 2019.

Turning to expenses. Non-GAAP operating expenses, which exclude compensation were $52 million compared to $47.4 million in 2019. The increase in operating expenses was primarily related to the Company's overall headcount growth and facilities expansion to support the advancement of Sangamo's therapeutic pipeline and manufacturing capabilities.

Moving to the balance sheet. We ended the quarter with $363 million in cash, cash equivalents and marketable securities.

Following the end of Q1, we received $350 million from Biogen from the sales of stock and the upfront license fee.

As Sandy mentioned earlier, we believe we have the balance sheet strength to take us through important R&D milestones, including the first potential filing of the BLA for SB-525 for hemophilia A.

Turning to 2020 full year guidance.

We are reiterating our previously shared financial guidance and anticipate non-GAAP operating expense, which excludes estimated stock compensation expense of $25 million to be in the range of $245 million to $260 million in 2020. At this time, we do not expect any material negative financial impact from COVID-19 to our operating expense guidance.

We will continue to monitor the situation and provide an update in the future. In the meantime, we will continue to be good stewards of capital.

I'll now turn it back to Sandy for closing remarks.

Sandy Macrae

Thank you, Sung.

This quarter marks an important milestone with the closing of the Biogen agreement, which both significantly strengthened our balance sheet and represents yet another vote of confidence in our highly differentiated zinc finger protein genomic medicine platform from a top biopharmaceutical company.

In these unprecedented times, I’ve observed tremendous resilience and adaptiveness from our employees. And this has kept our business moving forward, including ongoing business development discussions, continued research and technical operations in our laboratories, and continued partnerships with our clinical trial sites. We feel a great sense of confidence in our business and in our ability to weather the COVID-19 crisis due to our balance sheet strength, strategic investments in infrastructure, and to the proven plans that we've established to facilitate our rapid return to more normal operations as this crisis subsides.

Operator, we are ready for questions.


Thank you. [Operator Instructions] Our first question comes from Maury Raycroft with Jefferies.

Your line is open.

Maury Raycroft

Hi, everyone. Thanks for taking my questions.

First question is just on hemophilia A.

So, with the Phase 3 trial posted to, can you talk more about the design at this time, including dose, steroid use, and what estimates are on how long it's going to take to enroll the study?

Sandy Macrae

Maury, thanks for your question.

As you can imagine that the Phase 3 trial is under the control and communication of Pfizer, and they will give all announcements about the design of the trial. And we want to respect that relationship with them. Everything they have told us so far guides to the trial moving ahead as planned.

One of the advantages of our partnership with Pfizer is they're a global organization that can take the trials to where the patients are available and where the COVID impact is less.

So, we look forward to them sharing more information with you as the year progresses.

Maury Raycroft

Understood. And then, for Fabry, you guys had mentioned before that you had more patient screen failures than you initially expected.

Just wondering if you’ve implemented enrollment criteria changes, and have those been helping?

Sandy Macrae

Bettina, would you be able to talk to that?

Bettina Cockroft

Yes, of course. Hi, there.

So, we have been looking at implementing some changes. And as you will have noted from the communication today, we have actually enrolled patients into the Fabry study.

Now, of course, during the COVID pandemic, we have been very-cautious as to assessing the best timing, for dosing the first subjects, but we have had an uptake and that has resulted in inclusion of patients into the study.

Sandy Macrae

And Bettina, do you feel that the changes you’ve made to the protocol have permitted that and facilitated that?

Bettina Cockroft

Exactly, absolutely.

Maury Raycroft

Okay. And then, last quick one was just on -- wondering if you have formalized plans to conduct a renal biopsy for Gb3 reduction in this initial part of the study or could that come in later on? Maybe if you could just talk more about the plans.

Sandy Macrae

We haven't discussed our plans for the Gb3 and for renal biopsy.

As you can imagine, that is a complex issue about patient benefit and the risk of a renal biopsy.

We are very appreciative of the FDA trying to make medicines for Fabry, get to patients as quickly as possible by allowing registration quicker with the renal biopsy and are very aware of that and are incorporating it into our plans.


Our next question comes from Gena Wang of Barclays.

Your line is open.

Gena Wang

Thank you for taking my questions. I have two questions.

The first is regarding hemophilia A update, and second question regarding Fabry disease.

So hemophilia A update, I understand Pfizer emphasized the importance of 18 months data.

Given follow-ups on last ASH, is it fair to say 4Q this year likely will be a good timing to show data? And for the Fabry disease, you did mention you enrolled several patients.

Just wondering how many patients? And also, is this still possible to present initial data beginning of next year? Also, could you remind us the first dose for Fabry disease?

Sandy Macrae

Let me take the first question before passing you on to Bettina but warning you that we haven't talked about the first dose yet. But, if I could do the hemophilia A, unfortunately the world time moves at just the same rate and patients are only now coming out of their 18-month point, and that's very weak patients and it will take throughout the rest of the -- this and the next quarter for the majority of patients to reach their 18-month point.

So, Pfizer will lead all communications as part of the deal for a transition like this.

You agree which company will lead all communications, and that is in Pfizer's hands and they will decide when they have data that they will share with you all. I know this is frustrating. But, it has to be a single company that leads that.

And Bettina, can you talk about our enrollment in Fabry, please?

Bettina Cockroft

Yes, absolutely.

So, as far as Fabry is concerned, to address your last question first, we're going to be showing data after we've completed dose escalation across the three cohorts that we have in our protocol. We want to make sure that we present a mature data set that can represent the safety and efficacy of ST-920. And in terms of which doses we were planning to test, we have said low, medium and high.

We will disclose specific doses at an appropriate point in the future. What I can say is we've learned a lot about AAV6 through our SB-525 hemophilia A program and we've made protocol amendments to the Fabry program to take those learnings and also FDA guidance into account.

So, we look forward to updating further on this in the future.

Gena Wang

Okay. How many patients already have enrolled?

Bettina Cockroft

I think we’re not...

Sandy Macrae

Yes, exactly, we’re not disclosing that. But, we have patients enrolled.

We have interest from sites, and it's just a matter of us choosing when to dose the first patient. I'm sure you would agree that we need to choose wisely because the patients will have to come in for monitoring. And we want to make sure that they're safe and that the health service is not overstressed.

Gena Wang

Thank you.


Thank you.

Our next question comes from Whitney Ijem of Guggenheim.

Your line is open.

Whitney Ijem

Hey, guys. Thanks for taking the questions. I wanted to follow up on Fabry.

So, I guess first, can you give us any color on the entry criteria that were adjusted that kind of facilitated enrollment, just curious if we could learn more there. And then, the second question is on the endpoints.

You mentioned, you won't present data until you have a complete data set. I guess, what does that mean in terms of follow-ups and kind of what endpoints are you tracking, or is that the 12 months safety follow-up that’s reflected on clin-trials?

Sandy Macrae

So, Whitney, thank you for your question. The criteria that we adjusted were not things about antibody criteria, like gene therapy things. They were more our understanding of what Fabry patients look like, and the right patients to put into the study. Each time a company like ours goes into a new disease, we learn from the first few patients. And Bettina and her team have done an excellent job in simply understanding for patients are available.

When we see we won’t talk about the study until it's complete, what mean is that we've gone through the -- each of the dose cohorts. And as soon as we have biochemistry data from each of the dose cohorts, the low, medium and high as Bettina has said, we will share them with you.

You're absolutely right that there will be follow-on data that we'll look at additional parameters, including in some patients’ biopsy. But, we hope to be able to share the biochemistry initially and talk to you about the results of our intervention.

Whitney Ijem

Got it.

Just quick follow-up in terms of the biochemistry. What particular endpoints, I guess, are you looking at there? And what's the cutoff? I guess, is it like three months or six months of biochemistry you want to have at that higher dose to kind of see the threshold announcing the data?

Sandy Macrae

We haven't described that.

You've been with us for a long time, and you understand our cautiousness in speaking too soon and waiting for the results to stabilize.

So, as we can, most inform you and most inform the patient community.


[Operator Instructions] Our next question comes from Ritu Baral of Cowen.

Your line is now open.

Ritu Baral

I'm going to take another stab at the Fabry screening criteria question. Can’t help myself. I understand that there's different aspects to the disease, and as you said, like there have been other companies in Fabry gene therapy and they may be not optimized initial patients. And specific to Fabry, are you more interested in proving the effect in Fabry manifestation for kidney? Can you talk about how you think about unmet need in kidney versus unmet need in cardiac, maybe other organ systems, as you think about the disease, the patient you want and the unmet need across your key small molecule and some of the other gene therapies addressing those conditions?

Sandy Macrae

So, I'm good to let the Bettina talk to us in a moment. But, you can imagine this is first time any of us has moved to gene therapy into Fabry. And with the first few patients we learned about the effect to pharmacology, the plasma levels. And with the first few kidney biopsies, we’ve learned from those.

So, I think it would be premature for me to declare what is the right thing to do. We, last week or the week before, had three Fabry patients take part in a companywide Zoom conversation, talking about the things that still bother them. And it's clear that Fabry patients are not receiving all the benefits they need, and there's still a medical need. But Bettina, would you like to talk to that?

Bettina Cockroft

Yes, of course. And said, Sandy, we've had conversations with many experts in the field of Fabry to address exactly what you’re highlighting, kidney and cardiac and other organ being affected. We should point out at this point in our trial, this is a Phase 1/2 trial, its primary objective at this point is safety and tolerability of ST-920.

And so, we hope to see some of the safety and tolerability as we saw with the AAV6 based gene therapy SB-525. And moving on from there as we learn, we turn the focus on organs that make more sense moving forward to address that unmet need.

Ritu Baral

Got it. And then, Sandy, you mentioned, as you guys are moving forward with activities, you mentioned some additional regulatory meetings that you are going to engage with regulatory agencies with some of the new bandwidth that you have in COVID. Could you elaborate on what sort of regulatory meetings you might need? I mean, obviously Treg is moving into the clinic, Fabry is moving into the clinic. Can you give us a little color around that?

Sandy Macrae

So, can I make sure I understand your question? You are asking, are we having additional regulatory meetings with the agency or with agencies around COVID?

Ritu Baral

No, not around COVID, but in your comments initially around how there have been changes with work streams with COVID. It sounded like you had the opportunity to engage with regulatory agencies some more on programs, or I may have misunderstood you. But, I'd love to know what regulatory meetings are planned, say, in the next quarter or two on the program?

Sandy Macrae

So, I'm going to pass this to Bettina to help out on this one. And then, I'm going to make more general comments. Bettina?

Bettina Cockroft

Yes, absolutely.

So, you're absolutely right. We’ve mentioned this earlier on today. What we're focusing on mainly and you've picked it up is TX200, specifically where we are engaging with regulatory agencies, especially the various European ones in the various countries that we are initiating our TX200 study.

And so, we are going through all those regulatory approval processes for that study. And that study is receiving a lot of feedback, even during this pandemic. Many of the agencies are still working hard. But, that's exactly what we're engaging in.

Sandy Macrae

And then, on a more general basis -- but on a more general basis, we have not had specific comments with the agency, with the any agency about how COVID impacts our program. I sit on the Board of Bio and there we have a regular conversation about how companies work in some ongoing studies are having to make modifications to the protocol, modifications to clinic data, and the agency, in particularly FDA has been very understanding about that. Sangamo hasn't had to have those conversations, if that’s what your question is.

Ritu Baral

Got it. The last question, sorry. Sandy, you also mentioned that you may be considering other partnerships -- partnership opportunity. Can you talk to the programs now in your current lineup that you think would be most appropriate for a partnership versus Sangamo taking them forward?

Sandy Macrae

So, if you think of what happened with Biogen, there was -- one of those products, the tau for Alzheimer's that we've been speaking to you all for a while ago, 1 synuclein that we've spoken for three months, and the other 10 were things that none of us knew were in the Sangamo pipeline. There are things that we could partner that you are aware of. And we have no plans at the moment to further partner our existing assets in the pipeline that you think of Sangamo's. But there are also many other targets within the genome, even within the CNS space that are not part of our current go ahead portfolio, but the others are interested in.

Ritu Baral

Got it. Super helpful. Thanks for taking all the questions.

Sandy Macrae

Thank you very much.


Thank you.

Our next question comes from Jim Birchenough out of Wells Fargo Securities.

Your line is open.

Yanan Zhu

Hi. Thanks for taking the questions. This is Yanan dialing in for Jim.

So, first, a question on the Alzheimer's program in collaboration with Biogen. Wondering, have you decided what might be the optimal delivery route for that program, whether it's IV or intraperitoneal type injections? And also, secondarily, is there a milestone, the first milestone that you think you will be communicating around? So, just wondering the timeline, for example, a nomination of a candidate or a first IND, so any sense of when that might be? Thanks.

Sandy Macrae

So, a bit like what we've said for Pfizer, it will be Biogen to make this communication. But, I wonder if Adrian could comment routes of delivery and Stephane on milestones.

So, Adrian perhaps first?

Adrian Woolfson

Yes. Thanks, Sandy. And thanks for the question. We understand that we haven’t disclosed the route to delivery and that will -- we’ll leave it to Biogen to do that. But I will just say, for example, if you are referring ASGCT over this week, we'll be talking about some of our, attempts to derive new, serotypes using directed evolution and so on in our capsid library.

So, we've got an active program doing that. And obviously anything that comes out of those programs will be relevant to the work of Biogen. But, it's fact that we're considering a range of different delivery routes. But, at this point, wouldn't want to be more specific than that.

Sandy Macrae

And that's the presentation by…

Adrian Woolfson

Yes. That's right, Sandy, where we made these capsid libraries with different peptide insertions and insertion sites by using a range of different parental serotypes.

Sandy Macrae

And the way the contract is constructed.

Stephane Boissel

So, yes, Sandy, we closed the Biogen deal only a few weeks ago. And we have the on-boarding of the collaboration only a few days ago.

So, we need to be a little patient for the collaboration to really get started for the staying committee to meet before we can make any update as to the timeline on the various products we are meant to work on with Biogen.

Sandy Macrae

But the first shot of meeting was very successful I hear.

Stephane Boissel

Oh, it was. And despite very bizarre or unique setting because it was totally virtual and we had something like 80%, a combination of Biogen and Sangamo employees on the phone on Zoom as well. It was a real success, despite, again, kind of bizarre setting to start with.

Jim Birchenough

Thank you. That's very helpful. And then, just two quick questions regarding Fabry disease program.

Just wondering what is your competitive positioning against other AAV Fabry gene therapy approaches? And what's your impression from the initial biomarker data regarding enzyme that was presented recently? And whether in your low dose cohort, at starting dose cohort, you think to can achieve similar or higher in terms of enzyme level? Thank you.

Sandy Macrae

So can I just clarify your question? Are you saying in the second part of this -- who are you comparing against, a Freeline or bio, or Freeline.

Jim Birchenough

AAV, yes, Freeline.

Sandy Macrae

So, let me see if I can answer both of these. And none of us can claim that we have something different until we get into the clinic and see the results in patients. And therefore, that's why we're going to wait and complete the study at all three doses. I don't think it would be helpful for us to compare our low dose versus Freeline’s low dose, because all that matters is the final dose that you take into the clinic. And we are pleased with the design of our study. We're pleased with the animal results. And we're looking forward to showing clinical results.

And as Bettina emphasized, the study is about safety.

So, we will be looking at liver enzymes, we'll be looking at cardiac enzymes. We'll be making sure that this is as safe as possible, because only when you have that you can then start to compare on efficacy.

Jim Birchenough

Got it. That's very helpful. Thank you, Sandy.


Thank you. I'm showing no further question at this time. I'd like to turn the call back over to Sandy Macrae for any closing remarks.

Sandy Macrae

Thank you all for your interest. Thank you all for doing this virtually.

We are very fortunate at Sangamo to be able to proceed with a virtual team and our lab staff in the laboratories. And we look forward to sharing more results with you as the year progresses.


Thank you. Ladies and gentlemen, this does conclude today's conference. Thank you for participating.

You may all disconnect. Have a great day.