Good day, and thank you for standing by. Welcome to the Sangamo Therapeutics Third Quarter Earnings Call. At this time, all participants are in a listen-only mode. After the speakers' presentation, there will be a question-and-answer session. Please be advised that this call is being recorded. I will now turn it over to Louise Wilkie, Head of Corporate Communications and Investor Relations. Please go ahead.

Good afternoon. I'm Louise Wilkie, Sangamo's Vice President of Investor Relations and Corporate Communications. Thank you for joining us on the call today. On this call are several members of Sangamo's executive leadership team, including Sandy Macrae, Chief Executive Officer; Mark McClung, Chief Operating Officer; Prathyusha Duraibabu, Chief Financial Officer; Jason Fontenot, Chief Scientific Officer; Nathalie Dubois-Stringfellow, Chief Development Officer; and Bettina Cockroft, Chief Medical Officer. Slides from our corporate presentation can be found on our website, sangamo.com, under the Investors & Media section on 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 the therapeutic and commercial potential of our product candidates, the anticipated plans and timelines of Sangamo and our collaborators for initiating and conducting clinical trials, dosing and screening patients, and presenting clinical data, the advancement of our product candidates, advances with the preclinical programs to the clinic, our investment focus, and the sufficiency of our resources, our 2022 financial guidance, upcoming catalysts, 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, 2021, as supplemented by our quarterly report on Form 10-Q for the fiscal quarter ended September 30, 2022. The forward-looking statements stated today are made as of this date, and we undertake no duty to update such information, except as required by law. On this call, we discuss our non-GAAP operating expenses. A reconciliation of this measure to our GAAP operating expenses can be found in today's press release, which is available on our website. Now, I'd like to turn the call over to our CEO, Sandy Macrae.

Thank you, Louise, and good afternoon to everyone on the call. Sangamo continues to pursue scientific innovation and development excellence in the third quarter as we progressed our mission of turning groundbreaking science into potentially transformative genomic medicines for patients. I am pleased with the momentum we are achieving across our programs with our clinical data continuing to demonstrate the strength of our pipeline. Regarding our wholly-owned Fabry disease program, we were excited to present additional preliminary data from our Phase I/II STAAR study at three separate medical conferences since the last call. We continue to be encouraged by the promising data generated to date, showing sustained and elevated Alpha Gal activity along with our favorable safety profile as of the last data cut in July. These data demonstrate the potential of this therapy to replace the current standard of care and provide the potential for a one-time dosing alternative rather than a lifetime of enzyme replacement therapy infusions. I'm also pleased to share that this study has moved into the dose expansion phase, and we've begun dosing patients, including the first female patients in the study. We continue to make progress in our wholly-owned sickle cell program this quarter, dosing the sixth patient in the Phase I/II PRECISION study. This is the second patient to be dosed with our product candidate manufactured using improved methods. In addition, the FDA has granted Regenerative Medicine Advanced Therapy or RMAT designation to our sickle cell product candidate. Momentum continues behind our Phase I/II steadfast study of TX200 in HLA-A2 mismatched kidney transplantation. I am pleased to announce that the second patient was successfully dosed in late September, while the first patient has reached eight months post-infusion. Lastly, we jointly announced with Pfizer on September 22 that the Phase III APINE trial in hemophilia A has reopened recruitment. Trial sites resumed enrollment in September and dosing is expected to resume shortly. The resumption of this study is an important milestone as it takes us with our partner, Pfizer, one step closer to getting this potential treatment to patients in an area of significant unmet medical need. I'm proud of the team for the advancements we continue to make, staying true to our strategic focus on our research and development capabilities. Each incremental update brings us closer to our goal of creating transformative medicines for patients while creating long-term value for shareholders. I'd like to turn the call over to our Head of Development, Nathalie, who will discuss the data from our clinical programs in more detail. Nathalie?

Thank you, Sandy, and good afternoon to everyone on the call. We had several promising updates in the third quarter and are pleased with the progress being made across our clinical programs. In February, a fifth patient was withdrawn from enzyme replacement therapy in the Phase III STAR clinical study, evaluating Israel CEVA Parvovec, or ST-920, our wholly owned gene therapy product candidate for the treatment of Fabry disease. This marked the fifth and final patient in the dose escalation phase, who started the study on ERT to have been withdrawn from ERT. All subjects report they are not experiencing changes in symptoms and have not required resumption of ERT. After receiving endorsement from the Fabry Phase I/II study safety monitoring committee to progress to the dose expansion phase, five patients have been dosed in this expansion phase at the 513 be dose level, including the first two female patients in the study. Momentum also continues with additional patients currently screening and awaiting dosing, including more female patients. Since the last call, we were proud to present updated preliminary Fabry data at three medical conferences. In August, we presented data at the Society of the Study of Inborn Errors of Metabolism, SSIEM Annual Meeting in Freiburg, Germany, presenting data on five patients as of the February 14 data cut. Last month, we provided further updates at the 29th Annual Congress of the European Society of Gene and Cell Therapy, ESGCT in Edinburgh, Scotland, as well as the National Organization for Rare Disorders conference in Washington, D.C. At both events, we presented data as of July 21, 2022, presenting for the first time data across all four dose cohorts in the dose escalation phase. All nine treated patients exhibited sustained elevated alpha activity ranging from nearly two-fold to thirty-fold of the normal for almost two years of follow-up in the longest treated patients. All patients exhibited above-normal alpha-gal activity by five weeks after dosing. Importantly, patients in long-term follow-up maintain elevated alpha-gal levels for one year or more. We remain encouraged to see the durability of effect over this length of time. Also of note, four patients have undergone ERT withdrawal as of the cutoff date and continue to maintain elevated alpha-gal activity up to 28 weeks post withdrawal. The two subjects with substantially higher elevation in plasma lyso-Gb3 pretreatment show a 40% and 55% reduction from baseline in lyso-Gb3 level, respectively, after ST-920 dosing. Importantly, ST-920 continues to be generally well tolerated in the nine treated patients with no treatment-related adverse event higher than grade 1, except for one grade 2 event; no treatment-related serious adverse events were reported. Of note, no subjects have been treated with steroids, either prophylactically or reactively, and there have been no liver enzyme elevations requiring treatment. Since our last call, additional sites in this global study have been opened, including the first in the Asia Pacific, and we are actively preparing additional new sites. You can expect further clinical updates from the STAR study, including the first data from the expansion cohort in the first half of 2023. We continue to actively plan for a potential Phase III study and are engaging with health authorities, patient advocacy groups, and investigators. In the Phase I/II PRECISION study of BIVV003, a zinc finger nuclease gene-edited cell therapy candidate for the treatment of sickle cell disease, now wholly owned by Sangamo, we have dosed six patients. This is the second patient who has been dosed with a product candidate manufactured using an improved method shown in internal experiments to increase the number of long-term progenitor cells in the final product. We are pleased to continue to be making progress in the Phase I/II study and look forward to providing additional updates at an appropriate time. As Sandy mentioned earlier, on August 24, the FDA granted Sangamo's request for Regenerative Medicine Advanced Therapy, or RMAT designation for BIVV003, which is granted to regenerative medicine therapy intended to treat, modify, reverse or cure a serious condition for which preliminary clinical evidence indicates that this medicine has the potential to address an unmet medical need. The RMAT designation includes all the benefits of the fast-track and breakthrough therapy designation programs, including early interaction with the FDA. Furthermore, we were accepted to participate in a poster presentation at the upcoming American Society of Hematology or ASH Annual Meeting and Exposition, taking place in New Orleans, December 10-13, 2022. We look forward to presenting updated clinical data then. Finally, Phase III study design enabling activities and manufacturing readiness are in progress. Progress continues in the Phase I/II CETP study, our wholly owned TX200 CAR-Treg cell therapy candidate for the prevention of immune-mediated rejection in HLA-A2 mismatched kidney transplantation from a living donor. The second patient was dosed in late September, and I'm pleased to report that the product candidate continues to be generally well tolerated in both patients. Two control patients have been enrolled and transplanted, demonstrating ongoing interest in this study. We will provide further guidance on timing for dosing of the third patient when the transplant has been scheduled, and we have confirmed a potential dosing date. Finally, regarding the Phase III AFFINE trial, evaluating giroctocogene fitelparvovec, an investigational gene therapy for hemophilia A, we jointly announced with our partner, Pfizer, that the trial has reopened recruitment. Trial sites resumed enrollment in September, and dosing is expected to resume shortly. A pivotal readout is expected in the first half of 2024. In addition, with our partner, Pfizer, we've been accepted to participate in a poster presentation at ASH, December 10-13, 2022, providing updated clinical data on the Phase I/II ALTA study. We look forward to sharing updated data in December. I will now turn the call over to our Chief Scientific Officer for updates on our preclinical research program. Jason?

Thank you, Nathalie, and good afternoon, everyone. I'm pleased to report that we continue to leverage Sangamo's cutting-edge genomic engineering and cell therapy platforms to advance both wholly owned and partner programs towards the clinic. We are also making remarkable progress in our preclinical programs as well as expanding our proprietary genomic engineering toolkit and developing novel AAV capsids to enable more effective therapeutically relevant delivery of these tools. The Sangamo team had multiple presentations at this year's European Society of Gene and Cell Therapy Annual Conference, including work describing the use of our proprietary zinc finger nuclease platform to engineer both allogeneic healthy donor regulatory T cells and autologous paces. Additionally, data from preclinical studies performed with our collaborators at the Massachusetts Institute of Technology’s Broad Institute were presented at the International Prion 2022 Conference in Berlin, Germany this September. We were thrilled to hear the strong feedback from key opinion leaders at the conference who expressed their excitement about the science and its potential for patients in need. This work demonstrated the ability of Sangamo's engineered zinc finger protein transcription factors to specifically reduce prion protein expression in the brain and significantly extend survival in a mouse model of prion disease. We look forward to providing updates on this and other programs in the future. I will now turn the call over to our Chief Financial Officer, Prathyusha, for an overview of the financial results. Prathyusha?

Thank you, Jason, and good afternoon, everyone. Our detailed financial results for this quarter are available in the press release issued this afternoon, which can be found on our website. We ended the quarter with approximately $350 million in cash, cash equivalents, and marketable securities, which represents a net decline of $14 million from the prior quarter. This reflects our proactive approach to both prudent capital management and balanced capital raises in these uncertain markets and speaks to the continued investor interest in our company. We raised approximately $75 million in net proceeds under our after-market offering program since the beginning of the year. We continue to focus our investments in three key areas: advancement of our clinical programs, including Fabry, sickle cell, and TX200; progression of our preclinical programs and CNS pipeline; and optimization of our in-house manufacturing capabilities. Turning to our 2022 full-year guidance, we expect our full-year non-GAAP operating expenses to be lower than previously guided, and land between $280 million to $290 million. This range excludes estimated non-cash stock-based compensation expense of approximately $35 million. We expect a significant portion of our operating expenses to be invested in the continued advancement of our lead programs, including Fabry Phase III planning activities, Phase I/II activity for sickle cell disease and TX200, and preclinical work in CAR-Tregs and genome engineering indications in the central nervous system. I will now turn the call back to Sandy for closing remarks.

Thank you, Prathyusha. We are pleased with our achievements during this quarter and the progress we have made across our clinical and preclinical pipeline. We are a clinical-stage genomic medicine company, building momentum with our novel science, clinical execution, and in-house manufacturing. The progress this quarter takes us one step closer to delivering potentially transformative therapies for patients in need while creating long-term value for shareholders. I'm so proud of our team here at Sangamo who worked tirelessly to advance our programs and carry out our mission. We are delighted with the positive momentum and look forward to updating you in the near term with several expected milestones, including presentation of updated Phase I/II data in sickle cell disease, presentation at ASH of updated Phase I/II data in hemophilia A, additional Phase I/II Fabry data in the first half of 2023, including additional ERT withdrawal data and the first data from the expansion phase, and dosing of the third patient in the Steadfast Phase I/II study. At this time, we would like to open it up for questions. Operator, please open the line for questions.

Thank you. At this time, we will conduct a question-and-answer session. Our first question comes from Gena Wang with Barclays.

I have two questions. The first one is about the sickle cell data at the ASH and we all saw the abstract released earlier today. It seems like a placeholder, with a data cutoff of May 3. I'm just wondering, at the full data presentation, will we see two more patients at ASH? My second question is regarding the Fabry program. Regarding the expansion cohort data in the first half of '23, will you hear about the five-patient data? Or will we see more patient data beyond the current enrolled five patients? Additionally, what type of data will you be sharing? Will it mainly be on the biomarker data like Alpha-Gal, or what kind of clinical profile will be your goal to move to the next step?

Gena, thank you for your question. As I understand, you're asking a question about sickle and a question with Fabry. So I'm going to pass the sickle question over to Nathalie, and then Bettina will cover the Fabry question. Nathalie?

So patient 5 in the trial was dosed in May 2022, and it was the first patient to receive a product candidate manufactured using the improved process. We expect to share early preliminary data from this patient at ASH in December via poster presentation, along with an update of the first patient dose.

So Gena, thank you for the question around Fabry. As you have seen, we're very excited to be sharing that we have now dosed five patients in the expansion cohort. That brings us to a total of 14 patients dosed in the Fabry study, of which if you do the math, it means we have seven patients treated at the highest dose level. We do believe ST-920 has the potential to provide an alternative to the current standard of care. We will be sharing, as we pointed out, in the first half of 2023, data from both additional data from the dose escalation as well as the early data from the expansion cohort patients, and this will include biomarker data as well as additional clinical data, the exact nature of which we will share in the first half, so in early 2023.

Our next question comes from Luca with RBC.

Perfect. This is Lisa on behalf of Luca. Two questions for you today. First is on the data. I wanted to follow up on the baby data presented at ESGCT last month. I think we noticed there was at least one patient who withdrew from ERT but subsequently had an increase in the plasma alpha-activity after the withdrawal. How should we rationalize this response given you would expect that the alpha-gal levels to drop after the ERT withdrawal? My second question is on the Phase III planning. You reiterated today that planning is underway. Just wondering if you've had discussions with the FDA about what a Phase III could look like, what would be the primary endpoint and what you would need to show in terms of durability given this is a gene therapy approach.

Thank you for your questions and for your attention to our ESGCT presentation. I will pass this to Bettina, but I'll assist her a little with the second part. We've spent considerable time planning and thinking about the Phase III study. We have discussed it with the agency and will share the study details when the time is right.

Absolutely. Thank you so much for that question. On patients on ERT who have withdrawn from ERT, you asked about the Alpha-Gal activity expression. We expect Alpha-Gal activity to be sustained over time, and that's what we are seeing, not only in patients who were naive or pseudo-naive but also in patients on ERT who are withdrawn from ERT. That is the objective of our therapy. In fact, we believe that ST-920 has that potential to provide an alternative to the current standard of care of ERT. If you were referring to plasma lyso-Gb3 levels, the specific patients are all doing well. The plasma lyso-Gb3 remains within the range seen in patients on ERT. The investigators are happy with the data and the patients remain off ERT. I hope that addresses your question.

And I would encourage you to remember that this is a cutting-edge clinical science. These are the first patients from whom we have all gathered data with this treatment, the withdrawal of ERT and the early data that you've seen is with the lowest dose response. We hope we plan to be withdrawing high dose; the dose we've chosen to go forward into Phase III and show patients withdrawn from ERT with that. The other thing I would ask you to think about is even for patients on ERT when we added our gene therapy, they showed additional benefits. I think this is sometimes overlooked. Patients who have been on ERT for a long time, who then got gene therapy, reported feeling better, showing improvements in symptoms. This really speaks to the potential of our medicine.

Our next question comes from Nicole Germino with Truist.

This is Alex for Nicole. For the CAR-Treg platform, would you consider outlining or engaging in other collaborations with pharma? If so, could you provide some color on the types of partnerships that you would consider that might work best for Sangamo? Relatedly, what are your thoughts on big pharma dropping some IL-2 programs that we've seen in the news in autoimmunity? Does this change, or does this pave the way for CAR Tregs in the space?

Mark, you run our business development group. Would you want to talk to that?

Sure. Thanks for your question. We're excited with the progress that we're making with Tregs, where we believe we're the first company to have dosed a patient as we've communicated earlier. Our commitment is to continue to move forward with that study and drive it towards a proof of concept. If companies approach us, similar to what we've done in the past, if they can bring resources, know-how, and funding to accelerate the therapy to patients faster than we can, we will always evaluate that. But we're not commenting on any current conversations that we might be having in that regard.

And also on the IL-2 platforms, have you seen any commentary on that?

Jason, can you comment on IL-2 and where we fit in amongst the range of Treg programs?

Yes. Thank you, Sandy, and thanks for the question. I think the latest news that I'm aware of regarding pharma dropping an IL-2 program was related to the use of IL-2 in oncology, which is a different approach than some of the IL-2 muteins that are being explored to enhance Treg biology. Nonetheless, it is important to point out that while IL-2 muteins can be used to expand regulatory T cells and offer potential therapeutic benefits in autoimmune diseases, it's a fundamentally different approach because it relies on systemic delivery of IL-2 and increasing regulatory T cells throughout the patient's body. The approach we're taking with CAR T regulatory T cells is to target the regulatory biology and cells using the chimeric antigen receptor to the relevant tissue. And so that's a much different proposition, where the kind of immune regulation that regulatory T cells afford is directed in a specific and potent way towards the tissue where the autoimmune pathologies occur. Furthermore, the IL-2 muteins, despite best efforts, still have some reactivity with effector T cells and NK T cells. This complicates any interpretations of the effectiveness of those since hitting effector T cells can run the risk of exacerbating autoimmune diseases.

Thank you, Jason, for the insight.

Please stand by for the next question. Our next question comes from Yanan Zhu with Wells Fargo.

I have a question about Fabry and a question about the sickle cell program. So for the Fabry GCT presentation, it looks like the lyso-Gb3 levels increased in patients who discontinued ERT. Since that data cutoff is quite early in 2021-2022, it's been quite a few months since that data cut-off. You mentioned that nobody has resumed ERT. I was just wondering if we could interpret that to mean those patients have somehow stabilized their lab levels. Additionally, could you remind us about the criteria for resuming ERT?

Thank you for your questions. Bettina, can you provide clarification on that?

For sure. Thank you for that question. We were very pleased to present the latest Fabry data at ESGCT. I can confirm that we now have five patients who are off ERT, and that none of them have resumed ERT therapy. The investigators are happy with the data they are seeing, including the plasma lyso-Gb3 data. Regarding the criteria for resuming ERT, this is really dependent on a series of biomarker and clinical data and ultimately, a decision made by the investigator with the patient. So far, those patients are doing well.

I think the most important thing is this is at the investigator's discretion. The patients are doing well, and therefore, the investigators are comfortable keeping them off ERT. The levels you've seen are similar to what other patients on ERT would have for their lyso-Gb3.

That's very good to hear. For the sickle cell program, you discussed Phase III design and manufacturing readiness being underway. I was curious how much of that progress has been informed by what you saw in patient #5, the patient on the new manufacturing product? Also, do you plan to pursue this program by yourself, or how urgent is it for you to find a partner for this program?

Natalie, can you answer that?

We're currently focused on completing the Phase I/II study and progressing Phase III enabling activities. We're really excited to have this program in our hands, and we're planning to take this program forward, assuming the data continue to support a Phase III program. The next patient that has improved manufacturing will be very informative to see if we have a body of evidence to continue to Phase III. However, we're also planning ahead, and you'll have to wait for ASH poster to see a little bit more data.

Got it. And what about partnership opportunities?

Our intention is always to do what is best for the program. We're looking to gather data, and we won't see the full data set from the new improved process until the first half of next year. At that point, we'll make the necessary decision. We view it as whether we do it ourselves or a partner handles it; the important thing is getting a successful treatment to patients.

And for our next question, our next question comes from Greg Harrison with Bank of America.

This is Mary for Greg. Regarding Fabry, as you approach the trial, could you comment on the physician and patient efficacy interest in the gene therapy for Fabry and the impact of ERT freedom in this population? Additionally, what is considered clinically meaningful here?

Your line is hard to follow. Could I ask you just to repeat the question, please?

Yes, absolutely. As you approach the Phase III trial, could you comment on the physician and patient advocacy interest in the gene therapy and also the impact of ERT freedom in this population?

Mark, I think this is one for you.

Thanks for the question. As a general rule, we engage with physicians very early, actually prior to Phase I, and work with them to provide input into our protocol designs, but also to better understand patients. We also bring patients in to speak to us to share their experiences with disease so that our scientists and researchers can really understand what the patient requirements are. We're particularly proud of this effort and we believe it has allowed us to make great progress with the program. We'll provide next steps at the appropriate time.

Our next question comes from Kelly with Stifel.

This is Kelly on behalf of Ben Burnett. I just had one quick question regarding your preclinical programs in brain disorders, including prions and tau therapies, Huntington's, etc. Could you talk about these a little bit more in detail and maybe when we'll see them maturing into the clinical phase?

Thank you for your question. We'd love to share more about these. Unfortunately, in these partnerships, the communication is controlled by the partners. What I want to reassure you about is our team sits on steering committees, development committees, and research committees with the partners, and they're moving forward. We have a hidden early portfolio that is in our partners' hands. They're passionate about them and love the science of Sangamo. We hope to be able to share more soon as they reach milestones and progress towards the clinic. Did you also ask about prions? Jason, I know you're excited about the Prion results. Can you touch on that?

Yes. Thanks, Sandy. One of our scientists, Bryan Zeitler from Sangamo, presented at Prion 2020 earlier this year, data generated in collaboration with the Broad Institute at MIT, studying the ability of our zinc finger transcriptional repressors to be delivered to the brain of mice in a mouse model of prion disease and suppress the expression of prion protein, thereby preventing the spread of the disease. The data was remarkable. In comparison to control and other approaches tried in similar models, such as ASOs, our zinc finger transcriptional repressors potently reduced prion protein expression and dramatically extended the life of the mice in this model. Several key opinion leaders in the field were impressed with the data, with one even commenting that it was the most exciting data of the conference. We're very excited about this wholly owned program, and we're exploring moving it forward into humans as soon as feasible. There are complications since this is mouse data, and we are navigating next steps to prepare for an IND for testing in humans if feasible.

Our next question comes from Ritu Baral with Cowen.

I wanted to ask about the profile of the female Fabry patients that you've enrolled. Are they on the full ERT dose? I'm wondering about their kidney versus potential cardiac involvement or aspects of their disease. Furthermore, in this expansion cohort, is there a target number of female patients that you're looking to enroll to inform the Phase III? And then I have a follow-up.

Thank you for your question. It's exciting that we've moved into female patients. Bettina, can you provide more details?

Yes. We've detailed the patient characteristics, which you can find in our ESGCT slides. Patients have various characteristics, and we plan, per protocol, to dose up to 30 patients as part of the expansion phase, including both males and females. This also encompasses a renal cohort and a cardiac cohort. While we're not specifying the details of how many patients we've enrolled in those specific cohorts, we'll provide an update in early 2023 on that.

Just to clarify, the females could be in that cohort plus other females with varying conditions.

Could you comment on where you stand on CMC for the commercial product for the potential launch of the pivotal Fabry trial, especially regarding assays, validation of assays, and potency assays?

We prioritize CMC development equally to clinical aspects of the trial. I'm glad you've raised this, as the clinical piece often gets more media attention. However, the only way to develop these medicines effectively is by rigorously adhering to CMC and manufacturing protocols. We are on track with everything needed for Phase III and commercialization.

And can I squeeze one last question in? Can you elaborate, Sandy, on the biopsy schedule for patients in the expansion cohort?

Absolutely. For patients in the expansion cohort, we have two patients from cohort four who are naive patients for whom we have collected biopsy data. These are the earliest patients with kidney biopsy data. We plan to share that information in early 2023, and are also collecting kidney biopsies for patients who are naive or soon in the expansion cohort. We're grateful to the patients for agreeing to undergo biopsies, as they are not minor procedures. This includes both the entry biopsy and a follow-up, reminding us to appreciate the efforts of patients in drug development.

Are these going to be 12-month or 18-month biopsies?

These are six-month intervals for biopsies.

Please stand by for the next question. Our next question comes from Patrick with HC Wainwright & Co.

Just a couple of follow-ups. First on the Fabry program, could you discuss the safety and tolerability profile you've seen so far, and what it is about this profile that has enabled you to avoid some adverse events seen in other programs in the space? To what extent could these learnings be applied to current or future gene therapy or editing programs in development from your platform?

Thank you for your question. You really don't have much to say about the safety profile.

Thank you, Sandy. Yes, on the safety and tolerability side, as you've seen, we are doing exceptionally well. The patients are thriving. We have not needed to treat any of the patients with steroids reactively, and patients have not been treated with steroids prophylactically. We are continually monitoring these patients. We now have seven patients dosed at the highest dose cohort. What's key is that the clinical trial team has been attentive to details around patient education regarding the infusion schedule. We're pleased with the safety and tolerability profile.

We have used AAV6 across MPS trials, hemophilia trials, and now Fabry. The patient experience has proven remarkably well tolerated, even at the 513 level. While we observed occasional liver function test events in the hemophilia trial, those resolved quickly with steroids. Interestingly, we haven't seen any of these in the Fabry trial. That raises questions about why this is. The truth is we don't yet have enough experience to understand if the cargo makes a difference.

That's helpful. Regarding preclinical pipeline, with many programs in CAR-Treg cell therapies for autoimmune disorders and gene editing for neurological diseases, could you frame which program you think is underappreciated and when we could expect updates on underappreciated programs?

Jason, you're particularly excited about the CAR-Treg programs and the CNS indications; can you highlight which ones?

In the CAR Treg space, we've discussed our advancing programs targeting multiple sclerosis and inflammatory bowel disease. We view both areas as great opportunities for Treg therapeutics. We're excited about their advancement and the potential to revolutionize how autoimmune diseases are treated by directing immune regulation to specific tissues, such as the CNS for multiple sclerosis. Our zinc finger transcriptional regulators can upregulate or downregulate genes, and those are in collaboration with Biogen and Novartis in neurodegenerative and neurodevelopmental diseases. We're excited about the progress being made. The partnerships with these companies underscore the value of the toolkit we have. We have also advanced our internal programs, including Prion, alongside several other nondisclosed programs. Additionally, we've made remarkable advances in developing new AAV capsids that can deliver our zinc finger tools to the CNS, enabling us to explore more diseases.

That's helpful. Thank you very much.

At this time, I would now like to turn it back to Louise Wilkie for closing remarks.

Thank you. Thanks once again for joining us today and for everyone's questions. As a reminder, you can access the earnings release and 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.