Good afternoon and welcome to the Sangamo Therapeutics Third Quarter 2024 Teleconference Call. Please be advised that today's conference is being recorded. I would now like to turn the conference over to your speaker today, Louise Wilkie, Vice President of Investor Relations and Corporate Communications. Please go ahead.

Thank you. Good afternoon, everyone. Thank you for joining us on the call today. On this call are several members of the Sangamo executive leadership team, including Sandy Macrae, Chief Executive Officer; Nathalie Dubois-Stringfellow, Chief Development Officer; and Prathyusha Duraibabu, Chief Financial Officer. Slides from our corporate presentation can be found on our website sangamo.com under the Presentations page of the Investors and Media section. This call includes forward-looking statements regarding Sangamo’s current expectations. These statements include but are not limited to statements relating to Sangamo's cash runway, plans to obtain additional capital and ability to continue to operate as a going concern. The therapeutic and commercial potential of Sangamo's product candidates and technologies, Sangamo's ability to earn and receive payments from its collaboration and license agreements, including the Genentech and Pfizer agreements, Sangamo's expectations regarding new collaboration and license agreements, the anticipated plans and timelines of Sangamo and its collaborators for clinical trials, clinical data presentations and releases, regulatory submissions and regulatory approvals, upcoming catalysts and milestones and other statements that are not historical facts. Actual results may differ materially from what we discussed today. These statements are subject to certain risks and uncertainties that are discussed in our filings with the SEC, specifically in our annual report on Form 10-K for the fiscal year ended December 31, 2023, as supplemented by Sangamo's quarterly report on Form 10-Q for the quarter ended September 30, 2024 and subsequent filings and reports that Sangamo makes from time-to-time with the SEC. The forward-looking statements stated today are made as of today and we undertake no duty to update such information except as required by law. Please note that all forward-looking statements about our future plans and expectations are subject to our ability to secure adequate additional funding. Now, I’ll turn the call over to our CEO, Sandy Macrae.

Thank you, Louise and good afternoon to everyone joining the call today. As we near the end of 2024, I'm extremely proud of the progress we are making this year. We are committed to translating groundbreaking science into medicines that transform the lives of patients and families afflicted with serious neurological diseases. The updates we will share today demonstrate several meaningful advances towards that goal and show how we believe Sangamo is well positioned for continued progress. First, Sangamo has transformed from a Phase I/II company to a pre-BLA company as a result of significant regulatory developments in our Fabry disease program. Second, Pfizer continues to engage in discussions with regulatory authorities concerning our hemophilia A program, both of which have the potential to provide a long-term financial foundation for our core neurology pipeline. Third, we signed a neurology epigenetic regulation and capsid delivery license agreement with Genentech and received $50 million in upfront license fees and milestone payments. And finally, we submitted our first ever IND application for a neurology indication. These developments demonstrate that Sangamo is steadily executing upon our strategy, is driving potential medicines towards patients in need, and is continuing to advance plans to put the company on a more stable financial footing. Nathalie will share more context in a moment but I want to start out by highlighting the significant clinical and regulatory progress made this quarter in our Fabry disease program, following alignment with the FDA on a clear regulatory pathway to accelerated approval. This pathway reduces the time to potential approval by 3 years and avoids the requirement for an additional lengthy and costly registrational study which is incredibly impactful given the unmet medical need for patients with Fabry disease. We are delighted to have such a clear regulatory pathway that could bring this treatment to patients significantly sooner than originally anticipated and have begun to execute BLA readiness activities ahead of a submission anticipated in the second half of 2025. As you can imagine, this announcement has generated a lot of interest from external stakeholders, including patients who tell us about the remarkable change our treatment has brought to their lives, investors and potential strategic partners. We continue to engage in ongoing business development discussions as we seek to get this treatment to patients as quickly as possible. Following the recent top line readout from Pfizer for the Phase III AFFINE trial in hemophilia A, we also moved closer to potential regulatory submissions for this program which has the potential to unlock up to $220 million in regulatory and commercial milestones for Sangamo over the next 2 years. Pfizer will present a detailed Phase III data update at the upcoming American Society for Hematology or ASH Annual Meeting in December. And Pfizer has advised us that they are discussing this data with regulatory authorities which is very encouraging. Taken together, the Fabry and hemophilia A programs could provide Sangamo with a solid and long-term financial foundation for our core neurology pipeline. We are thrilled to be in the enviable position of seeing up to 2 potential BLA submissions in 2025 for product candidates developed by Sangamo, each of which leverage the best of our science and capabilities and address significant unmet patient needs and commercial opportunities. This exciting momentum propels our neurology pipeline forward as we work to advance our science and technology for neurological indications. This quarter, we signed our first neurology epigenetic regulation and capsid delivery license agreement. We have granted Genentech an exclusive license to our highly potent zinc finger repressors that are directed to tau, a critical gene involved in Alzheimer's disease and other tauopathies as well as an additional undisclosed second neurology target. For these same targets, we also granted Genentech an exclusive license to our industry-leading neurotropic delivery capsid STAC-BBB which has demonstrated potent blood-brain barrier penetration and brain transduction in nonhuman primates. We have received $50 million in upfront license fees and milestone payments from Genentech which extended our cash runway to allow us to continue advancing other ongoing business development activities. This agreement has further drawn interest in our science and enhanced our ability to attract new potentially valuable partnerships. We are currently advancing business development discussions with additional potential collaborators seeking to license our novel intravenous capsid, STAC-BBB and we believe this capsid is a potentially valuable source of additional non-dilutive funding. This quarter, we also submitted our first ever IND application for a neurology indication. We expect our lead program, ST-503 for intractable pain to advance into the clinic in the middle of 2025, assuming clearance of the IND with the FDA with our expected prion clinical trial authorization submission followed close behind by the end of 2025. Our cash runway remains unchanged and is sufficient to fund our planned operations into the first quarter of 2025, absent any potential funding from a Fabry partnership, hemophilia A milestone payments from Pfizer, or additional STAC-BBB collaboration agreements. Alongside the 2 anticipated BLA submissions next year, we see a viable path to financial stability. I would now like to hand it over to Nathalie, our Head of Development, who will share additional context and also take us through other pipeline updates. Nathalie?

Thank you, Sandy. I'm pleased to share that we announced last month that we have established a clear regulatory pathway for accelerated approval with the FDA for isaralgagene civaparvovec, or ST-920, our gene therapy candidate for Fabry disease. The FDA has confirmed that the estimated glomerular filtration rate (eGFR) slope data at 52 weeks from all patients in the ongoing Phase I/II STAR study can be used as the primary basis for approval under the accelerated pathway. This eliminates the need for an additional costly registrational study, which we had previously expected, and significantly shortens the timeline for potential approval by about three years. After analyzing the clinical data from the STAR study, which showed encouraging safety and efficacy results, we engaged with the FDA on alternative approval pathways. Renal issues, such as proteinuria or decreased eGFR, typically occur early in life for almost all male and many female Fabry patients and may lead to end-stage renal disease and early death. The eGFR is measured in milliliters of cleansed blood per minute per body surface area. For context, the average untreated patient shows an eGFR slope of minus 5.6. However, in the 18 patients treated with isaralgagene civaparvovec, who have over a year of follow-up data, we saw a statistically significant positive mean annualized eGFR slope. Achieving a positive eGFR slope is a notable accomplishment for Fabry patients, especially since other therapies tend to show improvements over untreated patients but still demonstrate a negative slope overall. Given this latest data, the FDA concurred that the eGFR slope at 52 weeks can be used as an intermediate clinical endpoint to support accelerated approval. The FDA also indicated that the eGFR slope at 104 weeks could be evaluated to confirm clinical benefits. The complete data necessary for the accelerated approval pathway will be ready in the first half of next year, paving the way for a potential Biologics License Application submission in the second half of 2025, which is three years earlier than we originally estimated. We are thrilled to have a clear regulatory pathway that could provide this treatment to patients much sooner than expected. Fabry disease is a serious condition with significant unmet medical needs, as highlighted during the National Fabry Disease Foundation Patient Conference I attended in October. Fabry patients discussed their difficulties with current treatments, including side effects, insufficient symptom relief, and breakthrough pain. They also mentioned the logistical challenges of long infusions every two weeks, which sometimes leads to missed doses. As a reminder, our Phase I/II STAR study has enrolled and treated 33 patients, capturing a wide range of Fabry cases. We have both ERT-treated and ERT-naive patients, as well as males and females, and those with cardiac or renal issues. With the positive annualized eGFR slope observed among these different Fabry patients, we expect that the BLA submission will encompass the entire Fabry patient population aged 18 and older. Also in the STAR study, all 18 patients who were on ERT at the study's start have successfully ceased ERT, with the first patient recently achieving three years off ERT, which is a significant milestone. More broadly, the longest-treated ST-920 patient has now reached four years of follow-up, and our data remain promising, with all patients maintaining normal or elevated levels of plasma alpha-Gal A enzyme activity. Regarding next steps, we are moving forward with BLA readiness activities and continuing discussions for potential collaborations. We are dedicated to working with the European Medicines Agency to identify the best regulatory path forward in Europe, and following a successful PRIME kick-off earlier this year, we are preparing for ongoing discussions. We hope to achieve alignment among regulatory agencies and expect to share an update early in 2025. Now moving to giroctocogene fitelparvovec, an investigational gene therapy we are developing with Pfizer for patients with moderately severe to severe hemophilia A. On December 9, Pfizer plans to present detailed data from the Phase III AFFINE trial at the 66th ASH Annual Meeting and Exposition. The ASH abstract released last week confirmed that the trial met its primary endpoint, demonstrating a statistically significant decrease in the total annualized bleeding rate from week 12 to at least 15 months post-infusion compared with routine Factor VIII replacement prophylaxis. Key secondary endpoints, including the percentage of participants with Factor VIII activity greater than 5% at 15 months and the annualized bleeding rate for treated bleeds, were also met, showing superiority against prophylaxis. Importantly, giroctocogene fitelparvovec was generally well tolerated with no study discontinuations. These findings further validate our commitment to genomic medicine aimed at improving patient lives. Partnering with a company like Pfizer, which has strong commercialization capabilities, is critical as we enter this partnership in hemophilia A. Pfizer has informed us that they are in discussions with regulatory authorities regarding the Phase III AFFINE data. We can earn up to $220 million in milestone payments from Pfizer upon achieving certain regulatory and commercial milestones, along with a 14% to 20% royalty on potential sales from this program if it is approved and commercialized. These advancements allow us to focus on our mission to treat debilitating neurological disorders with innovative genomic medicines. We believe our ability to integrate powerful zinc finger epigenetic regulation technologies with leading capsid delivery methods could enhance our neurology pipeline and transform the treatment landscape for indications where central nervous system therapy delivery has been difficult. This quarter, we submitted an IND application to the FDA for our Nav1.7 program, or ST-503, aimed at treating intractable pain. Neuropathic pain can arise from various conditions affecting the central or peripheral nervous system, such as surgical trauma, spinal cord injuries, nerve compression, neurological conditions, infectious diseases, and metabolic and hereditary syndromes. ST-503 is not designed for sporadic or acute pain but for chronic pain that profoundly impacts patients' lives over many years. Assuming FDA clearance of the IND, the Phase I/II study will evaluate ST-503's effectiveness in treating idiopathic small fiber neuropathy (ISFN), a painful condition affecting sensory nerves. ISFN has an estimated prevalence of at least 43,000 patients in the U.S., and more broadly, peripheral neuropathy affects nearly 40 million Americans. While various therapy options exist, including antidepressants, anticonvulsants, and opioids, there are no lasting or curative treatments available for ISFN, highlighting a considerable unmet medical need. There is substantial evidence linking sodium channels to the pathophysiology of neuropathic pain. ST-503 employs an adeno-associated virus vector carrying an engineered zinc finger repressor to specifically target the SCN9A gene, which encodes the Nav1.7 channel essential for pain signaling. Developing small molecules targeting Nav1.7 is challenging due to the structural similarities between sodium channels, which complicates selectivity and increases off-target effects. By directly targeting the SCN9A gene, ST-503 effectively reduced Nav1.7 sodium channel expression in sensory neurons in animal models and significantly alleviated hypersensitivity following a single intrathecal administration. Our preclinical research indicates that ST-503 is well tolerated in nonhuman primates, with notable reductions in Nav1.7 observed and no off-target effects. This preclinical data demonstrating ST-503's potential as a treatment for chronic neuropathic pain has been discussed in greater detail in a manuscript published in BioArchive earlier this quarter, and it is also accessible on our investor page at sangamo.com. We expect to initiate the Phase I/II study in mid-2025, marking a transformative step for Sangamo as it is our first neurology program to enter clinical trials. If efficacy is confirmed, we aim to expand the use of ST-503 to other populations suffering from various chronic neuropathic pain types. Moving on to our program addressing prion disease, we continue to advance the enabling activities for clinical trial authorization using our unique STAC-BBB capsid. Prion disease encompasses a group of conditions with critical unmet medical needs that we believe our technology can address. With over 1,500 new cases diagnosed each year in the U.S. and Europe, this rapidly progressive disease is invariably fatal, typically within 12 to 15 months after symptoms start, and there are currently no effective treatment options available. In October, we shared updated data at the Prion 2024 Conference showcasing the effectiveness of our zinc finger repressor in a mouse model of the disease across multiple dose levels. The zinc finger repressor significantly decreased prion mRNA and protein levels in the brain, improved survival rates in mice, and inhibited the formation of toxic prion aggregates. Additionally, findings from nonhuman primate studies presented at Prion 2024 illustrated that a single intravenous dose of the prion zinc finger repressor via STAC-BBB led to substantial and widespread repression of the prion gene in transduced neurons. We anticipate submitting a clinical trial authorization for this program in the fourth quarter of 2025. I will now turn it back to Sandy for closing remarks.

Thank you, Nathalie. In closing, we are delighted with the momentum being generated this year and are committed to the continued advancement of both our wholly owned and partnered programs. In 2025, Sangamo science could lead to BLA submissions for up to 2 separate gene therapy programs that we believe have the potential to fund the neurology company in the long term. We plan to dose patients in our first-ever neurology epigenetic regulation study. And we plan to submit an IND for the second study which would be the first-in-human study of our novel proprietary STAC-BBB neurotropic capsid. We are also currently engaged in advanced business development discussions for our new potential STAC-BBB collaborations. This is remarkable progress for a company of our size. We are pleased to have delivered these milestones in 2024 and plan to continue executing on our strategy in the coming months as we complete the task of transforming Sangamo into a neurology genomic medicine company. We see an exciting future as we make progress in addressing our long-term financing needs and look forward to building upon this year's progress in 2025 as we further advance our therapies to patients in need. Operator, please open the line for questions.

Our first question comes from Gena Wang from Barclays.

Congrats on multiple achievements here. So maybe I'll just focus on the Fabry program. Two related questions. The first one is regarding the FDA comments that eGFR slope at 52 weeks can serve as an intermediate and clinical endpoint. Does that mean FDA wanted to see statistical significance of the end of the 52 eGFR compared to the baseline? And then eGFR slope at the 104 weeks may be assessed to verify clinical benefit. Can you elaborate what does that mean? Do you need to continue to show statistically significant benefit compared to baseline? Or do you need to show positive trends continue at the 104 weeks? And the second question is related to the strategy. I think, Sandy, you mentioned that it has been a while since you wanted to seek partnership for this program. And now given the latest update, what is your latest thinking regarding this program? Do you think it will be most value generative for the shareholders? So would that be keep in-house or seeking partnership? If seeking partnership, what kind of evaluation will you be looking for?

Thank you, Gena, for the questions. So I'm going to pass to Nathalie. We haven't given details of the statistical analysis that the agency has asked us to do. It really is incredible that both the eGFR has shown to be a positive slope and also that the agency has embraced this and agreed to accelerated approval at 1 year. Nathalie, can you add some color to this?

Yes, thank you. We're pleased to have a clear regulatory pathway for accelerated approval that could expedite access to this drug for patients. The FDA has accepted that the data at 52 weeks can serve as the primary basis for approval under the accelerated approval program, utilizing the eGFR slope at that time across all patients. This represents an intermediate clinical endpoint that will facilitate accelerated approval. Additionally, it's important to note that there are no limitations on biologic products that receive accelerated approval. Moreover, the FDA indicated that data from the 104 weeks can be used to confirm clinical benefit. Therefore, we believe there is no requirement for any further studies. We will discuss this during the pre-BLA meeting with the FDA, and we expect to present the 2-year data for the 32 patients in 2026 for full approval.

Yes, Gena, this is quite important to get clear because I know that a number of people conflate accelerated approval with a confirmatory study. I want to be absolutely clear that no confirmatory study has been asked for or is required. We will submit the data for the 1 year, at which point there will be 32 patients at 1 year but also 19 of them will already have reached their 2-year point. We anticipate that we would be submitting the full 2-year data set a year later. The agency has said that they would recommend looking at this as confirmation of the 1-year data. What's been really interesting is when we've looked at the patients that have reached 2 years already is that the 1-year predicts the 2-year data. So the patients are positive at 1 year, it predicts that they'll also be positive at 2-year. So this is exciting for Sangamo because all of a sudden, the BLA filing has been pulled in 3 years. We're doing everything possible to drive this forward to make sure we get it to patients with a filing in the second half of next year. As to your other question about partnerships, as you can imagine, there are a number of people who have been very interested in these results. It's wonderful when we can announce a partnership around a quarterly call. Unfortunately, these things just take time and we've almost had to refresh the partnership discussions with this new data. We hope to be able to share more about it in the near future. But we want to do a deal that gets it to patients with a filing in the second half of next year and a launch sometime in the first half of '26. I hope that answers your question.

Our next question comes from Yanan Zhu from Wells Fargo.

This is Kwan on for Yanan. So our questions are also around the Fabry program. Can you share with us when may we see the next data update from the program?

Nathalie?

Yes. So now our Phase I/II has become a registrational study. So we are being very careful about disclosing the details of the data. And we expect to have the data in the second quarter of 2025 and share at that time the top line data.

So to be clear, the last patient joined this study in April of this year. The last patient's last visit will be in April of next year. It then takes some period of time to clean the data and that will allow us to submit the data in the second half of next year. Once we have that data cleaned and prepared, we will look for the best opportunity to share the data with all of you.

Got it. And some quick follow-up. So for patients with longer follow-up, you mentioned that they still have a positive eGFR slope. But do you see any change in the slope when patients' follow-ups get longer? And also in the ERT patients, do you see any change in the eGFR slope when patients withdraw their ERT?

So there are so many questions that I'm sure we would all like to discuss about this data, and we look forward to sharing it at the right time. What I can say is the longest-lasting patient in the study is over 4 years and 4 months. And those are the patients who are in the lowest dose of the dose escalation study and they're doing very well. The alpha-Gal continues to be produced at similar levels, which is very encouraging. There are so few patients at that time that to do an eGFR slope would not be wise until more patients get to the longer time points. Nathalie, anything?

Yes. And I can also confirm that the positive eGFR slope that we are observing at this point is across all patients, whether they started on ERT or they are naive or pseudo-naive, whether they are male or female.

Our next question comes from Maury Raycroft from Jefferies.

Congrats on the progress. As a follow-up to Gena's question earlier, I think you've mentioned you could include propensity match control data, too, as part of the filing package. Just wondering if you can comment on whether the FDA will put more weight on comparing versus the baseline or versus the propensity match control data. And can you talk about where you are at with getting the propensity match control data? Is that something that you think you will need in order to maximize potential with the partnership?

We cannot comment on how the agency will evaluate the various data components. However, throughout our discussions, they have stated that they consider the overall data. This includes the eGFR, evaluated as a group rather than as individuals, comparing data from baseline to one year and then to two years against historical trends for Fabry disease, where there typically is a drop of over five points in a year. Current knowledge about ERT agents indicates a reduction of between 1.5 and 2.5 over the same timeframe. The agency will also assess the sustained effects of alpha-Gal, the ongoing control of lyso-Gb3, and note that alpha-Gal levels in skin biopsies have significantly increased. They will review SF-36 scores, patient-reported pain, and FOS-MSSI. The entirety of this data set is very persuasive. It’s also crucial to remember that all 18 patients who started on ERT have since been removed from it and are doing very well. Therefore, while eGFR may be a key factor for the agency's approval, it is enhanced by the complete data set.

Got it. Yes, that all makes sense and it's helpful. And just wanted to clarify too for the milestone payment from Genentech for the $50 million. Did you receive all $50 million? Or was there $40 million recorded this quarter? And will you get an additional $10 million from the tech transfer? Just wanted to clarify on that.

Maury, this is Prathyusha. Yes, we've received all of the $50 million. Only a portion of it, the $40 million was reflected in our quarter-end balance which is what you're trying to bridge to.

Our next question comes from Luca Issi from RBC Capital. Prathyusha Duraibabu, Chief Financial Officer, responded that they have received the full $50 million. However, only $40 million of that amount was recorded in their quarter-end balance, which is the detail being clarified.

Obviously, congrats on the regulatory alignment. Maybe 2 quick questions here for me. On Fabry, at a high level, can you just talk about how your conversation with the FDA has evolved over time? I find it fascinating that the FDA asked AVROBIO to run a head-to-head trial versus Fabrazyme using renal biopsies as the primary endpoint, not too long ago versus it sounds like the FDA is telling you that a single arm looking at serum biomarker is now sufficient. So what has changed there in your view? Again, any color there would be much appreciated. And then maybe on hemophilia A, maybe in the context of the BioMarin commercial debacle, if you will, how confident are you that Pfizer really will put a lot of energy and resources behind the approval and the commercial launch of this therapy?

Important questions. I don't recall how many years ago AVROBIO engaged in discussions with the agency. Was it 2, 3, or 4 years back when they were utilizing preconditioning cell therapy to demonstrate a benefit of their medicine? At that point, there were several companies, including 4DMT, Freeline, and others, involved in the Fabry landscape. Fast forward, many of these companies have since exited the field. Another significant change has been Peter Marks' role at CBER, where he acknowledges the limitations on requirements for small populations of genomic medicines for rare diseases, and he aims to assist us all in finding a way forward. I believe what Sangamo has agreed upon with the agency reflects Peter's insights turned into a strategic plan for an effective treatment. Nathalie, would you like to outline the timeline of how we arrived at this point?

Yes. Earlier in 2024, we had a Type C meeting with the FDA where we agreed to conduct a Phase IIb study with 25 patients for the approval process. Following this, we obtained additional data from our STAR trial, showing a positive eGFR slope in 18 patients after more than a year and in 6 patients after two years. We had also scheduled a prime meeting with the EMA at this time. During this meeting, we shared the eGFR slope data, and the FDA representatives present expressed encouragement and recommended that we submit this data to them. Since we have RMAT designation, we opted for a Type B meeting to discuss our findings with the FDA, asking if we could use the positive eGFR slope data from the 18 patients at one year as the primary basis for approval under an accelerated pathway in our Phase I/II study, thus bypassing the initially planned Phase IIb study. The FDA supported our approach, and that's our current status.

And I should say, in all my many years of working with the FDA, I've never seen a written response so clear. When asked if we could use the 12-month eGFR for accelerated approval, they said, yes, we agree. So we are very pleased with that. I think it hopefully is somewhat of a precedence for other rare disease companies. They're trying to do their very best for patients that are in incredibly difficult circumstances. If I could switch to your second question which was your reflections on the BioMarin launch of ROCTAVIAN. We are in a relationship with Pfizer. And so we're always very careful to reflect what Pfizer has told us. And before each conference call, we agree with them what it is, how they would like their medicine to be reflected. We're very pleased with the abstracts they're going to be showing at ASH. And I would encourage you to look at the data there and read from Pfizer's language about their enthusiasm for this product. But they have told us that we can say that they're in discussions with regulatory authorities. And that, to us, suggests Pfizer's continued enthusiasm for this product.

Our next question comes from Ritu Baral from TD Cowen.

Sandy, since the FDA endpoint revolves around renal function, are they requiring or requesting any proportion or quantum of data from the pivotal data set be from female Fabry patients or patients with cardiac variants? And if the answer is no, would you still be able to have some of that prospective data either in the open-label portion or from some other prospective analysis for a likely label at some point? Just as the cardiac variant and cardiac involvement of the disease gains importance, it might be an important commercial tool to have talking to the broader patient population.

So our discussions with the agency have focused on Fabry disease as a whole, rather than differentiating between men and women or renal and cardiac issues. We've been analyzing data primarily related to eGFR due to the impressive results we've observed. We're also considering all other elements of the data set for our submission. Notably, our measurements of alpha-Gal from skin biopsies show an increase, and there has been an improvement in renal function. Additionally, patients' reports on sweating and their well-being, reflected in the FOS-MSSI, indicate an overall enhancement in their conditions. Therefore, it stands to reason that the treatment should also have a positive impact on the heart and other areas. The agency is only requesting data from the 32-patient group, and there won't be more patients added for the Sangamo clinical development program. However, patients are eager for this treatment to be available to them as soon as possible. Your team recently met with the Fabry support group; would you like to share what insights were gathered from that meeting?

Yes. We were at the National Fabry Disease Foundation Patient Conference about 3, 4 weeks ago, and we held a patient group forum where we had patients that were on ERT, but we also had 3 patients that had received our gene therapy. And these patients are really very happy to have made that decision to enter the trial. We have patients that report that they were working with a cane. They now have dropped their cane. They're working normally. They feel so much better, "patients feel like it has a new lease on life." We have patients that have reduced pain. We have patients that now are taking jobs in the heat in the airport, carrying luggage and they're sweating and they feel great. So it's a general well-being that we're hearing from the patients. The patients that are on ERT are not very happy about their treatment. First of all, it's very burdensome. Every 2 weeks you get a 5 to 6 hours infusion. Often, you have someone coming at home, you have to organize this. It's pretty invasive. And really, what we've heard from the patients is after 10 days of their ERT, they start feeling not well. They're starting to have symptoms coming back and they still have to wait. And they have other medications that they're taking around the ERT pre or post. So it's really not a very good treatment from the patient side. And because it's burdensome, we know that there is a large population of Fabry patients that do skip doses because of the burden of ERT treatment.

Yes. We are very clear that ERT was a great advance in the treatment of Fabry. And we feel that this is now the next stage in that journey where they'll be able to get a single infusion of AV, extremely well tolerated with no serious adverse events related to the treatment and show this benefit over time. And it's a privilege to work on such a medicine and we look forward to finding the right partner to take this forward to get it to patients.

The question-and-answer session is now closed. I'll now turn it back over to Louise Wilkie for closing remarks.

Thank you and thank you once again for joining us today and for all of your questions. As a reminder, you can access our presentation on the Investor Relations section of the Sangamo website. We look forward to keeping you updated on our future developments. Thank you.

Thank you for your participation in today's conference. This does conclude the program. You may now disconnect.