Sangamo Therapeutics, Inc. Aktie

Good afternoon, and welcome to Sangamo Therapeutics First Quarter 2026 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, Head of Investor Relations and Corporate Communications. Ma'am, 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 Nikunj Jain, Interim 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, Sangamo's plans to obtain additional capital and its ability to continue to operate as a going concern, the therapeutic and commercial potential and value of Sangamo's product candidates and technologies; Sangamo's ability to establish and maintain collaborations and strategic partnerships, including for its Fabry disease program, the anticipated plans of Sangamo and its collaborators for clinical trials, regulatory submissions and regulatory approvals and other statements that are not historical fact.

Actual results may differ materially from what we discuss 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, 2025 and the quarterly report on Form 10-Q for the fiscal quarter ended March 31, 2026, 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. In the short time since our fourth quarter earnings call, we have continued to advance the rolling submission of the BLA for ST-920 in Fabry disease and made progress across our differentiated neurology pipeline. Nathalie will provide a detailed update on our development pipeline shortly.

This quarter, Sangamo's common stock transitioned to trading on the OTCQB Venture Market following the receipt of a delisting determination from NASDAQ Capital Markets due to noncompliance with NASDAQ's minimum bid requirements. We intend to appeal the determination at a hearing before NASDAQ in June. Our stock began trading on the OTCQB Venture Market earlier this month under the same ticker symbol SGMO.

While we are disappointed by the NASDAQ delisting determination, the transition to the OTCQB market will allow Sangamo to maintain access to the public markets while we seek to advance our promising development pipeline. We remain focused on pursuing a number of opportunities to raise additional capital, including evaluating all strategic options to maximize the value of Sangamo's assets.

Throughout this process, we have been and continue to be supported by a global investment bank. We continue to engage in business development discussions for a potential Fabry commercialization agreement, and we'll share more information as appropriate.

Before I hand over to Nathalie, I'd like to share an update from Boston, where I just returned, where we had 3 abstracts accepted at this year's American Society of Gene and Cell Therapy Annual Meeting. ASGCT is an important forum for showcasing innovation in gene and cell therapy, and I was proud to see our talented scientists present our most recent advances in zinc finger epigenetic regulation, specifically for Nav1.7 and prion disease, alongside a platform presentation on our Modular Integrase technology.

We have now achieved impressive levels of targeted integration in a variety of different cell types, moving us one step closer to our aspiration of achieving large-scale genome engineering to integrate large sequences of DNA into the genome.

I would like now to hand over to Nathalie. Nathalie?

Thank you, Sandy. First, I am pleased to share updates from our registrational Phase I/II STAAR study evaluating isaralgagene civaparvovec, or ST-920, our investigational gene therapy for the treatment of adults with Fabry disease.

As outlined during our recent fourth quarter earnings call, the rolling submission of a BLA to the U.S. FDA seeking approval of ST-920 under an accelerated approval pathway is in progress with the first 2 modules submitted. The team is actively advancing the activity required for the CMC module, and we anticipate completing the BLA submission as early as this summer, subject to our ability to secure adequate additional funding.

As a reminder, ST-920 has a clear pathway to accelerated approval agreed with the FDA using mean annualized eGFR slope at 52 weeks across all 32 dosed patients in the study. The FDA also affirmed to us in a very recent interaction that an additional confirmatory study will not be required and that we may submit 104-week data across all those patients when seeking traditional approval.

In February, we also presented detailed clinical data via 4 platform and poster presentations at the 22nd Annual WORLD Symposium that took place in San Diego, California. We continue to believe that the data support the potential of ST-920 as a one-time, well-tolerated and durable treatment to provide meaningful multi-organ clinical benefit that could fundamentally shift the Fabry treatment paradigm.

All 4 presentations are available on the Sangamo website.

Moving to our neurology pipeline. We have 6 clinical sites activated for the Phase I/II STAND study evaluating ST-503, our investigational epigenetic regulator for patients with intractable pain due to small fiber neuropathy or SFN. These sites are working to identify patients. This quarter, we were also pleased to have a manuscript published in Science Translational Medicine detailing the preclinical safety and pharmacology of ST-503 in human neurons, mice and nonhuman primates.

Finally, moving to ST-506, our epigenetic regulator for the treatment of prion disease. This quarter, we continued to advance clinical trial application, or CTA, enabling activities. The good laboratory practice, or GLP, toxicology study has been completed and analysis is ongoing.

In addition, we held a further productive interaction with the U.K.'s Medicine and Healthcare Products Regulatory Agency, or MHRA, including alignment on diagnostic testing, analytical validation and nonclinical safety matters.

I would like now to hand back to Sandy for closing remarks. Sandy?

Thank you, Nathalie. In closing, we have made good progress this quarter. The rolling BLA submission for ST-920 remains in progress with the first 2 modules submitted and with real clarity on the regulatory pathway reaffirmed very recently by the FDA. Six sites have been activated in the Phase I/II STAND study, evaluating ST-503 for patients with chronic neuropathic pain.

We held a productive meeting with the MHRA, including further alignment on key matters for the prion program. And we showcased continued progress across our pipeline and platforms at ASGCT, including a platform presentation showing impressive advances in our MINT integrase technology.

This remains an important period for Sangamo as we continue to advance our pipeline while actively pursuing strategic opportunities to strengthen our financial position and maximize the value of our assets. We will continue to provide relevant updates when we are able to.

Operator, please open the line for questions.

[Operator Instructions] Our first question comes from the line of Murray Raycroft with Jefferies.

This is James on for Maury. Congrats on getting the reaffirmation from the FDA on the 104-week endpoint. Given that the last patient reached the 2-year time point around April, can you walk us through the gating factors for analyzing that data set and disclosing the data set? Would you wait to present the 104-week data at a medical conference such as WORLD next year? Or could it be disclosed earlier after you file the BLA?

So as you know, we're -- I'm going to say, 3/5 the way through the rolling submission and the clinical module with the 1-year data is already submitted. We have a series of cleaning processes to gather the full 2-year data, and we'll work out once we have that when the best place to submit it. But I could -- we can reassure you, Nathalie, that we're now seeing patients out to 5 years' worth of experience, and it's looking very stable.

Yes. We absolutely show durability in the benefit for the patient at 5 years and beyond. We have now 4 patients that are beyond 5 years and with supraphysiological levels of alpha-Gal and safety of the product also remains remarkable.

And the 17 patients that are off ERT, some of them are over 3 years off ERT now?

Yes, absolutely.

Our next question comes from the line of Yanan Zhu with Wells Fargo.

This is Kuan on for Yanan. So with FDA's recent affirmation that 2-year eGFR data can serve as a confirmatory evidence, can you talk about how that will affect your BD discussions? And any updates on the BD discussion around the Fabry program that you can share with us?

I hope you can understand that we can't talk about those conversations. I am certain that the people who have been looking at this asset are pleased by the recent affirmation from the agency. Most importantly is to get accelerated approval as quickly as possible, and that's with the 1-year data.

Equally important is agency signaling that a second study will not be necessary because that would be expensive and delay the process. And as we now gather the 2-year data, not just for the eGFR, but for all the other benefits to the patients, we will plan when we can submit it. And I can assure you that the data is looking good. It's showing durability, and we look forward to sharing this at the appropriate time.

And I would now like to hand the conference back over to Louise Wilkie for closing remarks.

Thank you once again for joining us today and for 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.

This concludes today's conference call. Thank you for participating, and you may now disconnect. Everyone, have a great day.

Good afternoon, and welcome to the Sangamo Therapeutics Fourth Quarter and Full Year 2025 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, Louis Like, Head 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; Natalie DuBrastringfellow, Chief Development Officer; Nikunj Jain, Interim Chief Financial Officer; and Greg Davis, Head of Research and Technology.

Slides from our corporate presentation can be found on our website, sangamo.com, and under the Presentations page of the Investors & Media section. This call includes forward-looking statements regarding Sangamo's current expectations. These statements include, but are not limited to, statements related to Sangamo's Cash runway. Sangamo's plans to obtain additional capital and its ability to continue to operate as a going concern.

The therapeutic and commercial potential and value of Sangamo's product candidates and technologies; Sangamo's ability to establish and maintain collaborations and strategic partnerships, including for its Fabry disease program, anticipated plans of Sangamo and its collaborators for clinical trials, regulatory submissions and regulatory approvals and other statements that are not historical fact.

Actual results may differ materially from what we discussed today. These statements are subject to certain risks and uncertainties as are discussed in our filings with the SEC, specifically in our annual report on Form 10-K for the fiscal year ended December 31, 2025, and subsequent filings and reports that Sanco makes from time to time with the SEC. The forward-looking statements today -- 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, Louis, and good afternoon to everyone joining the call today. Sanga will continue to make significant pipeline progress in 2025 and into the first quarter of '26, set against a backdrop of regulatory and market uncertainty and with limited cash resources, in June, we announced positive top line results from the registrational STAR study in Fabry disease, including a positive mean annualized estimated glomera filtration rate or eGFR slope at 52 weeks across all those patients in the study which is the U.S. FDA reiterated in October 2025 may serve as the primary basis of approval under an accelerated approval pathway. .

The rolling submission of a biologics license agreement or BLA to the FDA, is in progress, seeking ST-920 approval, Sangamo's first ever wholly owned BLA submission. We transitioned to become a clinical-stage neurology company with 6 clinical sites activated in the Phase I/II STAND study in chronic neuropathic pain.

In April, we continue to demonstrate that we're a collaborator of choice for neurotrophic capsids with the announcements of a third neurology capsid license agreement, this time with Eli Lilly to deliver genomic medicines for up to 5 central nervous system disease targets. And we have raised over $130 million in funding since the start of 2025 through non-dilutive license fees and milestone payments as well as equity financing.

These are important achievements and I would like to sincerely thank everyone at Sage for their hard work, dedication and continued focus on our mission to help patients in need. I would now like to hand directly over to Natalie Dubo Stringfellow, our Chief Development Officer, to provide recent business updates from our prioritized pipeline.

I will then close the call by summarizing the key broader business takeaways from this quarter. Natalie?

Thank you, Sandy. First, I am pleased to share updates from our registrational Phase I/II STAAR study evaluating Izaragalcian Civaparovec, or ST-920 our investigational gene therapy for the treatment of adults with Fabry disease.

In December, we were happy to initiate the rolling submission of a BLA to the U.S. FDA seeking approval of ST-920 under an accelerated approval pathway. Rolling submission allows for a completed module of the BLA to be submitted and reviewed by the FDA on ongoing basis rather than waiting for the entire BLA to be submitted at once.

We have now submitted both the nonclinical and the clinical modules to the FDA. In addition, the antibody assay companion diagnostic, which is designed to screen patients for eligibility with ST-920 has been submitted to and accepted by the FDA Center for Devices and Radiological Health or CDRH, seeking premarket approval.

These are a significant milestone for Sangamo and for Fabry patients in need. I would like to sincerely thank everyone at Sangamo involved for their significant efforts in getting us to this point. The next key milestone is completion of the chemistry, manufacturing and controls, or CMC module.

We are very pleased to have completed manufacturing and testing of the process validation lots with acceptable results achieved and to have completed method validation. We are also excited to have manufactured our first commercial lot. We are working hard to advance the remaining required activities and anticipate completing submission of the BLA as early as this summer, subject to our ability to secure adequate additional funding.

In February, we presented detailed clinical data via 4 platform and poster presentation in the 22nd Annual World Symposium that took place in San Diego, California. We believe this encouraging data continue to demonstrate the potential for the endogenous production of alpha-Gal activity following ST-920 administration to transform the Fabry treatment landscape.

With a positive mean annualized eGFR slope at 1 year across all those patients in the study and at 2 years for 19 patients, we continue to see improved kidney function which marks a notable departure from the historical renal decline characteristic of the disease. The stabilization in cardiac function including stability of cardiac structure and cardiac biomarkers is also especially encouraging given that cardiovascular disease is the most common cause of death in Fabry disease patients and we were pleased to give a platform presentation dedicated entirely to this important topic, showcasing new cardiac specific data.

In addition to a well-tolerated safety profile, the ability to withdraw from enzyme replacement therapy and a range of other clinical benefits. These data continue to demonstrate how ST-920 shows potential as a onetime durable treatment option for Fabry disease that could fundamentally shift the current treatment paradigm.

At world, we also presented platform presentation on pharmacology and immunogenicity outcomes from the STAR study alongside a fertility embryo fetal development, AAV integration and germline transmission risk study in mice. All 4 presentations are available on the Sangamo website.

Next, I'd like to focus on our prioritized neurology pipeline. As in December, we were thrilled to receive Fast Track designation for ST-503, our investigational epigenetic regulator for Fabry with -- for patients, sorry, with intractable pain due to small fiber neuropathy or SFN.

As a reminder, Fast track designation aims to facilitate the development and expedite the review of new therapeutics that are intended to treat serious or life-threatening conditions and that demonstrate the potential to address unmet medical needs.

Company granted this designation are given the opportunity for more frequent interaction with the FDA. This clinical program may also be eligible to apply for accelerated approval and priority review if relevant criteria are met. SFN is a debilitating chronic pain disorder with limited effective current treatment options. So this designation underscores the high unmet patient need in SFN and the urgency to develop safe and effective non-opioid treatment alternatives.

Since the last update, we have activated an additional 4 clinical sites in the Phase I/II STAND study evaluating ST-503 to achieve a total of 6 active sites. These sites are working on identifying patients. Last week, we were also pleased to have a manuscript published in Science Translational Medicine detailing the preclinical safety and pharmacology of ST-503, in human neurons, mice and nonhuman primates.

This promising results provide the preclinical foundation for our Phase I/II STAN study. Finally, moving to [ ST-506 our epigenetic regulator for the treatment of prion disease to be delivered intravenously using our neurotropic STAK BBB capsid.

This quarter, we continue to advance clinical trial application or CTA enabling activities. The good laboratory practice or GLP toxicology study has been completed and analysis is ongoing. I would like to hand back to Sandy to provide a broader business and financial update. Sandy?

Thank you, Natalie. In closing, in times of regulatory change and careful financial management, we are pleased with the pipeline progress we have made since the start of 2025. A positive top line reduction our Fabry disease program, alongside continued productive engagement with the FDA have resulted in a rolling submission of the BLA for ST-920 with the first 2 modules submitted.

We became a clinical stage neurology company with the initiation of the Phase I/II STAND study in and we received fast track designation from the FDA for this program. And we have continued to show Sangamo as a collaborator of choice for neurotrophic capsids with announcements of our third STACK BDB license agreement.

These are important advancements. However, we will not be satisfied until we solve our long-term cash runway securing a commercial partner for Fabry remains our #1 focus, and we continue to engage in Fabry business development discussions with multiple potential partners.

Like me, I know many of you feel frustrated that this process is taking so long. Discussions of this nature are complex and facilitating the extensive due diligence required by potential partners takes time as they assess the regulatory environment for gene therapies.

We also continue to seek ways to raise additional capital, including an assessment of all strategic options for each of our assets and are in discussions with multiple potential partners, including alongside our focused efforts to secure a Fabry commercialization partner.

We will share more information as soon as we are able. Operator, please open the line for questions.

[Operator Instructions] while we can file the Q&A roster. Our first question comes from Maury Raycroft with Jefferies.

This is James on for Maury. Just to start off, can you provide more color on the revised timing for the Fabry BLA submission? What PPQ and other CMC-related activities are the primary gating factors here? And separately, I know you just touched on the Sandy, but can you comment on the status of the Fabry partnership discussions, whether the same counterparties previously engaged remain in dialogue with Sangamo and whether recent leadership changes at Zebra. I've had any impact on those discussions.

Thank you, Maria. So an important set of an important set of questions. So when we were given guidance that we could file for accelerated approval with a single study, the clinical data time lines were very clear because the patients were already in the study.

We then had to hustle to complete the CMC activity that would normally be performed over Phase II and Phase III.

And so the CMC has always been the piece that has been on the critical path for our filing. We've also had a number of very, very helpful interactions with the CMC group at the FDA. And most recently, they've given us very clear detailed pages of guidance on what was needed to complete the submission and maximize its chance of success. And of course, we are following those to the letter and making sure that we do all the right things for CMC, which is often the piece that is most challenging for cell and gene therapies.

And then there's a third bit that we haven't spoken about as much, which is we are managing our cash very carefully. We're managing our spending, and we want to ensure that our runway gives us the best possible time to fulfill a Fabry partnership.

And that wise prudent spending compared -- combined with the agency's requirement has just led to the time line for the CMC being a little longer than it previously was. You asked about the partnerships. We have been talking to people for or 18 months, a variety of people.

Most of the ones we spoke to last year have now gone. And many of the times, it was because of regulatory uncertainty. We are now speaking to multiple partners and are having good conversations with them.

But these are -- these 3 are new to the discussions. And so we need to go through the process of deep diligence, management presentations and then negotiations. And Murray, trust me, the team are doing everything possible to find the right partner and get this to patients when we are so close to a filing of a medicine that is going to fundamentally change the way Fabry patients are treated.

[Operator Instructions] Our NEXT question comes from Patrick Trucio with [indiscernible] .

Hi, everyone. This is Louis Santos in for Patrick.

I was wondering if you have had any additional interactions with the FDA recently or have any additional FDA actions planned, specifically relating to the acceptance all the eGFR slope as a primary endpoint?

And if anything has changed given the recent FDA changes.

Thanks, Lis. So we spoke to the agency last in October of last year and feel that they reiterated that the eGFR at 1 year could be used to file for accelerated approval. .

We haven't gone back to them. It's always a question of fulfilling what they've asked and submitting it, and we're now halfway through the rolling submission for -- we've submitted the clinical module and the preclinical module. We've submitted companion diagnostic, the CMC is in process, and then there's a final piece that kind of pulls it all together and gives us the full summary.

So we haven't -- we don't plan to go to the agency again because we're already partway through the process. If the agency changes or their way of dealing with gene therapy changes, we would, of course, address that. But at the moment, we feel we have enough guidance from to move ahead.

Thank you so much for the detail.

Thank you. I'm showing no further questions at this time. We do have a question from Luca Issi.

This is Kate Value. On Prime disease, maybe quickly, you mentioned that this is your first in-human trial for SECBcapsid. Does that time line also account for your partnered programs? -- that PBB's clinical proof of concept will be -- the prime 1 will be the first seen and your partners entrants into clinic or behind that? And related to it, when we look at which programs have the STACK-B taps it, is there an aspect of the approach that makes it more suitable for some indication versus others? -- or the decision to use the intrathecal [ AAV9 ] for neuropathic pain, for example, is just a matter of timing.

So I'm not sure I understood I heard all the parts of that, but let me try and answer what I think you were asking. You -- I think you asked why did we use AAV9 for neuropathic pain. And that program was started 4 or 5 years ago before the STACK BBB capsid was known and AAV9 is very effective when given intrathecally at dosing the Dorsoootgangline.

So it would be the logical choice. And AAV9 has been used and being seen to be safe and effective. Our STACK BBB, we are very pleased, and we're very pleased to have such imminent neurological companies choosing STACK BBB and taking it forward.

They are fortunate to have great resources, and we are working with each 1 of them to help them to understand the capsid and take it forward. And we feel we have good relationships with all of the 3, and we're encouraged by their enthusiasm to move their programs forward.

Thank you. And I'm showing no further questions at this time. I would now like to turn the call back to Luis Wilky for closing remarks.

Thank you once again for joining us today and for 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.

This concludes today's conference call. Thank you for participating. You may now disconnect.

Good morning, and welcome to Sangamo Therapeutics Third Quarter 2025 Conference 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, Head of Investor Relations and Corporate Communications. Please go ahead.

Thank you. Good morning, 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; Greg Davis, Head of Research and Technology; Prathyusha Duraibabu, Principal Financial Officer and Principal Accounting Officer.

Slides from our corporate presentation can be found on our website, sangamo.com, and 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, Sangamo's plans to obtain additional capital and its ability to continue to operate as a going concern, the therapeutic and commercial potential and value of Sangamo's product candidates and technologies, Sangamo's ability to establish and maintain collaborations and strategic partnerships, including for its Fabry disease program, the anticipated plans and time lines 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 fact. Actual results may differ materially from what we discuss 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, 2024, and our quarterly report on Form 10-Q for the fiscal quarter ended September 30, 2025, 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 morning to everyone joining the call today. This quarter, we continue to advance our clinical and preclinical pipeline while managing our cash resources carefully.

In September, we presented promising detailed clinical data from our registrational STAAR study in Fabry disease, demonstrating the potential for ST-920 as a onetime durable treatment of the underlying pathology of Fabry disease for all types of Fabry disease patients.

And in October, we were pleased to hold a meeting with the FDA to discuss the proposed efficacy and safety data package for a planned BLA submission where in the meeting minutes, the FDA reaffirmed its October 2024 agreement to use eGFR slope as an endpoint to support an accelerated approval pathway.

A particular highlight for me this quarter was attending the 15th Annual Fabry Family Education Conference that brought together more than 200 Fabry patients, family members and volunteers to provide educational presentations and gather insights from patients, including those who have received ST-920. This is an event we are privileged to attend each year, and it was humbling to spend time with this group of inspirational people of all age groups to learn more about their experiences with Fabry disease.

I found it striking to hear firsthand the challenges these patients face, including their experiences with currently available treatments, yet also see the hope they have for treatment breakthroughs and their strong understanding of scientific advances and their unwavering support for one another.

I came away from the event more convinced than ever that patients are seeking an alternative to current standards of care alongside the peaks and troughs and associated symptoms that they can bring.

Gene therapy, where the alpha-Gal A enzyme expressed all day, every day by the liver, is a fundamentally different treatment modality to what is available today. I was lucky to meet with some patients who received ST-920 as part of the STAAR study, and their enthusiasm and excitement was overwhelming. They couldn't wait to tell me their experiences, and one patient approached me to share how ST-920 has completely transformed their life. We have a responsibility to bring this medicine to the Fabry community.

In our prioritized neurology pipeline, we are excited now to be recruiting and enrolling patients in the Phase I/II STAND study in chronic neuropathic pain, our first-ever neurology clinical study following the activation of the first 2 clinical sites.

We also continue to advance our prion program ahead of the planned CTA submission next year.

I would like to now hand directly over to Nathalie Dubois-Stringfellow, our Chief Development Officer, to provide additional details on these important programs. Prathyusha Duraibabu, our Principal Financial Officer, and I will then close the call by summarying the key business and financial takeaways from this quarter. Nathalie?

Thank you, Sandy. First, I am pleased to share updates from our registrational Phase I/II STAAR study evaluating isaralgagene civaparvovec or ST-920, our investigational gene therapy for the treatment of adults with Fabry disease. This quarter, we presented encouraging detailed clinical data at the ICIEM 2025 Conference in Kyoto, Japan.

As we have shared previously, after a single dose of ST-920, a positive mean annualized estimated glomerular filtration rate or eGFR slope of almost 2 was observed at 52 weeks across all 32 patients dosed in this study. Furthermore, a positive mean annualized eGFR slope of 1.7 was observed in the 19 patients who have achieved 2 years of follow-up.

Supportive mean annualized eGFR slopes were also observed across a variety of patient subgroups, including gender, baseline ERT status, Fabry disease type and baseline eGFR, showing consistency in effect across Fabry patients in the study.

For the first time, we share cardiac data, including stable cardiac morphology, stable cardiac function and stability in early marker of cardiac damage in the 32 patients with at least 52 weeks of follow-up. These data are encouraging, particularly given that cardiac disease is a leading cause of death in Fabry disease patients.

As we have outlined previously, a range of key secondary endpoints were also positive. We continue to see strong durability in the study, up to 4.5 years for the longest treated patient, and we are pleased to see an encouraging ongoing safety profile. Indeed, every day that passes, we accumulate more data. And as of today, we are pleased to have 3 patients with at least 4.5 years of follow-up.

We believe that these data demonstrate the potential of ST-920 to provide meaningful and long-lasting clinical benefit to a wide range of Fabry disease patients, even above current standard of care.

In October, we held a meeting with the FDA to discuss the proposed efficacy and safety data package ahead of the planned BLA submission. We are pleased that the FDA meeting minutes reiterated the October 2024 agreement that we may use eGFR slope as an endpoint to support an accelerated approval pathway, agree on the adequacy of the safety package to support the BLA submission and provided valuable input on the clinical data package.

We continue to prepare for our anticipated BLA submission under the accelerated approval pathway planned for as early as the first quarter of 2026.

Next, I'd like to focus on our prioritized neurology pipeline, as this quarter, we've commenced patient enrollment and recruitment in the Phase I/II STAND study evaluating ST-503, our investigational epigenetic regulator for patients with intractable pain due to small fiber neuropathy or SFN, following the activation of our first 2 clinical sites.

SFN is a truly debilitating chronic neuropathic pain impacting more than 650,000 people across the U.S., Europe and Japan. We are thrilled to be recruiting patients for our first-ever neurology genomic medicine clinical study and expect to dose the first patient in the coming months.

This quarter, we are also pleased to present updated nonclinical data at the 9th International Congress on Neuropathic Pain in Berlin, Germany, which demonstrated the durability, potency and selectivity of ST-503 in nonhuman primate alongside a favorable safety profile. We believe the preclinical data for this program is compelling, and we look forward to seeing how this translates into humans.

We believe chronic pain is an area of strong market potential, and we're particularly encouraged by the FDA's recent draft guidance on the development of non-opioid analgesics for chronic pain, which seeks to accelerate safe and effective non-opioid treatment and to reduce prescription-related opioid misuse. We stand with the FDA in seeking safe, effective alternative pain relief options.

As a reminder, this is a dose escalation study, which, if positive, could allow us to broaden into other potentially high-value indications such as trigeminal neuralgia or oncology-related chronic pain.

Nav1.7 is a well-proven target with human genetic validation, which we believe provide pain franchise potential.

Finally, moving to ST-506, our epigenetic regulator for the treatment of prion disease to be delivered intravenously using our neurotropic STAC-BBB capsid. This quarter, we continue to advance clinical trial application or CTA-enabling activities for the program, ahead of our expected CTA submission as early as mid-2026.

Following on from our productive meeting with the U.K.'s MHRA earlier this year, where we aligned on nonclinical safety and the clinical study design, this quarter, we were pleased to hold another productive interaction with the MHRA, this time to align on the planned chemistry, manufacturing and controls, CMC strategy for the anticipated CTA submission.

In November, we presented updated preclinical data at the Prion 2025 Conference, which demonstrated the potent combination of epigenetic regulator and capsid delivery technology for the treatment of prion disease, including a profound survival extension that was observed in an aggressive mouse model of prion disease alongside widespread brain delivery and significant prion reduction in nonhuman primates.

Based on this compelling preclinical data, we are preparing for the CTA submission to test 506 in the clinic. Given the rapidly deadly nature of prion disease with no currently available treatment option, we anticipate demand for this planned study to be high with a short time frame to an expected data readout.

Furthermore, this study would mark the first-in-human testing of our STAC-BBB capsid, which, if successful, could unlock a broader neurology pipeline for advancement, including with potential partners.

We have deliberately chosen 2 lead neurology assets that use different delivery mechanisms and bring different development risk. We believe both of them independently offer significant potential value and provide additional expansion opportunity into related neurological indications.

I would like now to hand over to Prathyusha Duraibabu, our Principal Financial Officer, to provide a financial update. Prathyusha?

Thank you, Nathalie. This quarter, we continue to diligently prioritize and control spend while seeking ways to extend our cash runway as we continued business development discussions for a Fabry commercialization agreement. In October, we received $6 million upon Pfizer's exercise of a buyout option from our 2008 license to use zinc finger modified cell lines. We also continue to engage in business development discussions across our Sangamo pipeline and platforms.

As of today, we believe our cash and cash equivalents, including the license fee received from Pfizer and proceeds from sales of common stock under our at-the-market offering program since September 30 will be sufficient to fund our planned operations into the first quarter of 2026.

Before handing back to Sandy, I'd like to emphasize that we remain resolutely focused on solving our long-term funding foundation to advance our [ promising medicine ] pipeline. In the near term, we seek to bridge to that future through a balanced approach, actively pursuing non-dilutive business development opportunities while exploring appropriate capital options.

I will now hand it back to Sandy for closing remarks. Sandy?

Thank you, Prathyusha. To close, I'm pleased with the pipeline advances this quarter. We held a meeting with the FDA where in the meeting minutes, the FDA reiterated its October 2024 guidance that we may use eGFR slope as an endpoint to support an accelerated approval pathway. We presented detailed data from our registrational STAAR study in Fabry disease, including a positive mean annualized eGFR slope at 52 weeks, and we're pleased to observe a wide range of other positive secondary endpoints.

Taken together, we believe in the potential for ST-920 to provide meaningful, multi-organ, clinical benefits above current standards of care. The importance of this data has also been recognized externally with the acceptance this week of 3 platform presentations on our Fabry disease program at the upcoming WORLDSymposium in 2026.

This quarter, we began recruiting and enrolling patients in the Phase I/II STAND study for chronic neuropathic pain following the activation of the first 2 clinical sites. We expect to dose the first patient in the coming months. And we continue to advance our prion program towards an anticipated CTA submission as early as mid-2026.

As Prathyusha outlined, solving our long-term funding needs remains our #1 priority. We continue to seek ways to raise additional capital alongside our focused effort to secure a Fabry commercialization partner.

Before closing for questions, I want to reflect on the recent Nobel Prize in Physiology or Medicine awarded to Mary Brunkow, Fred Ramsdell and Shimon Sakaguchi for their research on regulatory T cells or Tregs.

At Sangamo, we proudly advanced the clinical development of Tregs being the first known company to dose a patient with an engineered CAR-Treg. In August of this year, we presented the clinical data from our TX200 study in kidney transplantation at the World Transplant Congress in San Francisco, showing the clinical potential of CAR-Tregs to create a tolerogenic environment in the kidney, alongside the ability to taper immunosuppression post TX200 dosing. These are encouraging results that we believe demonstrate the potential for engineered CAR-Tregs in organ transplantation. And we really want to say that we are thankful to everyone who has been involved in this first-in-human study.

While we are pleased with these innovative scientific advances, we remain focused on our promising neurology pipeline and look forward to sharing further novel scientific developments with you all.

Operator, please open the line for questions.

[Operator Instructions] Our next question comes from Gena Wang from Barclays.

This is Hang Hu from Barclays on behalf of Gena Wang. Just a couple of questions on Fabry disease. Could you share with us what's the latest progress in your partnership deal negotiation? And also on the regulatory front, you shared your progress with FDA. But in light of recent news from CBER about uniQure, is there any read-through to your Fabry disease drug program?

So I can't comment on the discussions uniQure had. What they're doing is important and difficult, and we simply wish them well for patients with Huntington's disease. But what I can tell you about is our interactions with the agency and the written meeting minutes that we have that confirm our ability to use the eGFR data for 1 year to get accelerated approval.

And when we look at our data, it's the eGFR is very clear and compelling, but it's the overall body of data, the safety of the product, the ease of use, the cardiac data, the SF-36, the kidney benefit. And I believe that's what the agency see, and that's why they have remained steadfast in endorsing the use of eGFR at 1-year to allow us accelerated approval and to file this next year.

We have also had other interactions with the agency on CMC and manufacturing. And I know those sometimes aren't quite as exciting as the clinical path forward. But for genomic medicines and for AAV manufacturing, having the agency's agreement on how you will manufacture it and what the packaging that is going to be required for approval is, is essential. And so we are very confident on that progress. And just to kind of reflect on that, now that the agency has reaffirmed our accelerated approval pathway, this can only be helpful for our business development discussions.

Yes. How about the first part of this question? Like could you share with us any progress and your status in partnership negotiation?

I'll just repeat what I said at the end there that now that the FDA has reaffirmed both the CMC and the clinical pathway, it can only be helpful in those discussions.

Our next question comes from Maury Raycroft from Jefferies.

Just kind of following up on the last question. Wondering if you're planning to have any additional meetings with FDA prior to a pre-BLA meeting? And do you need to have -- do you plan on having a pre-BLA meeting? And what additional clarification do you need from FDA at this point?

Nathalie, can you answer that?

Yes. We -- as a result of the meeting we had with the FDA, we're really pleased with the clarity on the clinical and safety package required for the BLA submission. As Sandy mentioned, we also have clarity on the element for the CMC strategy. And this interaction were recent, and we are exploring at this point if there are further topics that require a pre-BLA meeting.

Nathalie, this is a bit of a regulatory fine point. One doesn't have to have a pre-BLA meeting if you feel all the questions have been answered.

Absolutely. And because we have RMAT, we've had this year many interactions, either through meetings or correspondence where we had really good clarity on many of our questions. So at this point, we're really discussing internally if there is anything that remains to be discussed. But there is the pre-BLA...

The RMAT process works well, doesn't it, because it allows that ongoing interaction.

Exactly. Exactly. Yes, we were very pleased this year on the responses from the FDA, and they were always on time, and we've had no delays in our discussions.

Got it. That's helpful. And I guess just to -- for the BD partners, assuming that this is kind of a 3-way conversation where you're relaying the regulatory information to these BD partners. Is there any additional clarity that you need on the regulatory front that the BD partners need in order to make a decision?

I don't believe there are any questions left to answer. We have clarity on the primary endpoint and the agency has seen our 1-year data. So that was part of the reason we had that recent clinical meeting is because the last time we showed it to them, there was 18 patients at 1-year, and now there's 32 patients at 1-year and 19 patients at 2-years and the certainty of the eGFR slope remains. We have which is something that the partners -- potential partners would clearly want to know. And the CMC and manufacturing route is clear and the manufacturing process is underway and locked and loaded.

Our next question comes from Wells Fargo.

This is Kuan-Hung for Yanan. So our question is also around the Fabry program. Can you share with us in your engagement with FDA, has the topic of Commissioner's National Priority Review Voucher ever been mentioned? And do you think that could affect your BD discussions?

Nathalie, can you comment on that?

Yes. No, we have not discussed specifically that, but we are looking into it.

Got it. And have you shared all the details regarding the FDA meeting minutes with your potential partners? And any color you can comment on their reactions?

So the -- you can imagine when we got the minutes, we were pleased to share them with our partners or to share the essence of them. And any partnership that moves ahead, the partners would definitely see the minutes as part of the process. So they always get to see all of the regulatory correspondence as part of any business development deal.

Our next question comes from Patrick Trucchio from H.C. Wainwright.

This is Luis Santos for Patrick. First, I want to say it is great to see the progress and the extension of your runway is commendable and reflects your financial discipline, and we appreciate that.

I want to ask a couple of questions on 503 in neuropathic pain and regarding the STAND study and the dosing that you said is going to start in the coming months. Are there any additional challenges that we should consider recruiting this patient population? And looking ahead into the readout in about a year, what would be a clear win for -- to move this program forward? And I have a follow-up.

Nathalie, can you comment about how we're doing with getting the study going?

Sure. So we're pleased to have already 2 sites activated that are actually right now screening patients and looking at their -- the population. We've also broadened the population to SFN, not just idiopathic small fiber neuropathy, which really allows a broader scope in terms of finding patients. So we are very optimistic that we will be able to dose the first patient in the coming months.

We're also very active in opening other sites. We are going to have up to 10 sites in the study. And in terms of the success of the trial, of course, this is a dose escalation to look at the safety and tolerability of the study, but we're also looking at efficacy in term of the pain reduction from baseline using the PI-NRS, which is the overall Pain Intensity Numerical Rating Scale, which is broadly recognized and widely used as a preferred scale for pain measurement. And we will be monitoring this in a blinded fashion throughout the dose escalation.

Nathalie, it's been interesting the number of patients that have spontaneously from other places written to us and tried to get into the trial. It's clear that there's a high unmet medical need here.

Yes, absolutely. People have reached out to Sangamo or have seen the site activated in the clinicaltrial.gov page and have been directly contacting site to see if they could be eligible throughout the country.

And Luis, to your other question about what does efficacy look like, this is the first ever clinical dosing of something like this. And I think we will understand it with every patient that we dose. It's not a medicine that is for every patient with neuropathic pain because it's a once-and-done treatment that will change their pain sensation forever. And therefore, we will understand the population better in the coming months as we dose these patients.

And regarding your platform, can you discuss any ongoing partner interest in the STAC-BBB capsid? Any additional deals that could extend your runway? And also with your MINT platform progressing?

We're delighted with the progress of the capsid and the -- so yes, we are always talking to new people about the capsid. What's also important is the people that are working with the capsid, Genentech, Astellas, Lilly are also pleased and our relationship and discussions with them are going well. Greg, how is MINT doing?

MINT is doing well. So we continue to advance that platform in ways where we have minimal spend. So we're trying to derisk that technology from a molecular standpoint for MINT design towards genomic targets. And we're trying to use our money wisely in that to keep that moving forward. And we continue to advance business development negotiations for potential MINT partnership and look forward to sharing more information on those when we're able to.

Yes. It's -- the success of the integration with the MINT platform is increasing every time the team come and show me it. And it really is a fundamentally important piece of science that we hope to speak about more once in the future and hopefully put in the hands of others and Sangamo to make medicines from.

This does conclude the question-and-answer session. I would now like to turn it back to Louise Wilkie for closing remarks. The floor is yours.

Thank you once again for joining us today and for 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.

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