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DATE

Thursday, November 6, 2025 at 4:30 p.m. ET

CALL PARTICIPANTS

• Chief Executive Officer — Sandy Macrae
• Chief Development Officer — Nathalie Dubois-Stringfellow
• Head of Research and Technology — Gregory Davis
• Principal Financial Officer and Principal Accounting Officer — Prathyusha Duraibabu
• Head of Investor Relations and Corporate Communications — Louise Wilkie

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TAKEAWAYS

• ST-920 Clinical Data – Positive mean annualized eGFR slope of nearly 2.0 observed at 52 weeks in all 32 patients; A mean annualized eGFR slope of 1.7 was observed in 19 patients with two years of follow-up, Cardiac function remained stable among 32 patients with at least 52 weeks of follow-up.
• Durability of Effect – Three patients have at least 4.5 years of follow-up on ST-920 as of Q3 2025, supporting the safety and durability profile reported.
• FDA Regulatory Progress – FDA meeting minutes in October reaffirmed use of eGFR slope as an endpoint for potential accelerated approval, with agency agreement on safety and CMC packages for BLA submission.
• Fabry BLA Timing – BLA submission for ST-920 under the accelerated pathway is planned for as early as Q1 2026.
• Neurology Pipeline Progress – Patient enrollment and recruitment initiated in the Phase 1/2 STAND study for ST-503 targeting chronic neuropathic pain, with two clinical sites activated and dosing expected in the coming months.
• ST-503 Nonclinical Data – Preclinical results demonstrated durability, potency, and selectivity in nonhuman primates, alongside a favorable safety profile presented at a major neuropathic pain congress.
• ST-506/Prion Disease Platform – ST-506 advancement continues, with updated preclinical data showing survival extension, widespread brain delivery, and prion reduction; CTA submission expected as early as mid-2026.
• Strategic Business Development – $6 million was received from Pfizer’s buyout option in October 2025, with business development discussions ongoing for Fabry and other pipeline assets.
• Cash Position – Cash and cash equivalents, including proceeds from recent license and stock sales, are expected to fund operations into 2026.
• Pipeline Recognition – Three platform presentations on Fabry disease program accepted for the 2026 World Symposium, signaling external validation.

SUMMARY

Sangamo Therapeutics (SGMO 16.29%) reported regulatory confirmation from the FDA enabling submission of ST-920’s BLA for Fabry disease based on eGFR slope, with management planning to file as early as Q1 2026. ST-503 entered patient enrollment in its first neurology study for chronic neuropathic pain, with additional site activations ongoing. Updated preclinical findings for ST-506 in prion disease revealed survival benefits and broad brain distribution in animal models, establishing momentum for a mid-2026 CTA. Management identified financial discipline as a core operational focus, with capital planning and business development tied directly to advancing the prioritized clinical pipeline.

• CEO Macrae said, “the agency has reaffirmed our accelerated approval pathway,” attributing regulatory clarity as a key factor in partnership negotiations.
• Dubois-Stringfellow noted, “we’re really pleased with the clarity on the clinical and safety package required for the BLA submission.”
• Clinical investigators observed a consistent eGFR benefit with ST-920 across subgroups, including baseline ERT status, gender, Fabry disease type, and eGFR, in the STAR study, with cardiac stability also reported.
• Recruitment for ST-503 targets up to 10 sites, broadening to small fiber neuropathy beyond idiopathic cases to increase enrollment opportunities.
• Companies currently evaluating Sangamo’s delivery platforms include Genentech, Astellas, and Lilly, per direct discussion on partnership activity.
• Efforts to secure a commercialization partner for Fabry asset remain ongoing, enabled by regulatory agreement and encapsulated in ongoing business development activity.

INDUSTRY GLOSSARY

• eGFR slope: The rate of change in estimated glomerular filtration rate, a key measure of kidney function over time, used as a clinical endpoint in Fabry disease trials.
• BLA (Biologics License Application): Regulatory submission required for approval to market a biologic product in the United States.
• CMC (Chemistry, Manufacturing, and Controls): The FDA’s requirements regarding the methods and controls used in the manufacture of drug products.
• SFN (Small Fiber Neuropathy): A type of neuropathic pain condition involving damage to small sensory fibers, leading to chronic pain.
• RMAT (Regenerative Medicine Advanced Therapy): An FDA designation intended to expedite the development and review of regenerative medicine therapies.
• STAK BBB capsid: Sangamo’s neurotropic viral capsid platform engineered for targeted delivery across the blood-brain barrier.
• PI-NRS (Pain Intensity Numerical Rating Scale): A standardized tool used to assess pain intensity in clinical studies.

Full Conference Call Transcript

Louise Wilkie: Over to your speaker today, Louise Wilkie, Head of Investor Relations and Corporate Communications. Please go ahead. Thank you for joining us on the call today. Good morning, everyone. On this call are several members of the Sangamo executive leadership team, including Sandy Macrae, Chief Executive Officer; Nathalie Dubois-Stringfellow, Chief Development Officer; Gregory Davis, Head of Research and Technology; Prathyusha Duraibabu, Principal Financial Officer and the Principal Accounting 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 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 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 12/31/2024, and our quarterly report on Form 10-Q for the fiscal quarter ended 09/30/2025, 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.

Sandy Macrae: Thank you, Louise, and good morning to everyone joining the call today. This quarter, we continued to advance our clinical and preclinical pipeline while managing our cash resources carefully. In September, we presented promising detailed clinical data from a registrational STAR study in Fabry disease demonstrating the potential for ST-920 as a one-time, durable treatment of the underlying pathology of Fabry disease for all types of Fabry disease patients. 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 fifteenth Annual Fabry Family Education Conference. It 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 in associated symptoms that they can bring. A fundamentally different treatment modality to what is available today. Gene therapy where the alpha GalE enzyme is expressed all day, every day by the liver.

I was lucky to meet with some patients who received ST-920 as part of the STAR study, and their enthusiasm and excitement were 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 1/2 STAND study, our first-ever neurology clinical study, following the activation of the first two clinical sites in chronic neuropathic pain. 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 summarizing the key business and financial takeaways from this quarter. Nathalie?

Nathalie Dubois-Stringfellow: Thank you, Sandy. First, I am pleased to share updates from our registrational phase 1/2 STAR study evaluating Isaragal gene cvaparvovec 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 two was observed at fifty-two 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 two 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 and effect across Fabry patients in the study. For the first time, we share cardiac data showing stable cardiac function, including stable cardiac morphology, and stability in early markers of cardiac damage in the thirty-two patients with at least fifty-two 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 four and a half 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 three patients with at least four point five 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 standards 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.

This quarter, we commenced patient enrollment and recruitment in the Phase 1/2 STAND study evaluating ST-503, our investigational epigenetic regulator for patients with intractable pain due to small fiber neuropathy (SFN), following the activation of our first two clinical sites. SFN is a truly debilitating chronic neuropathic pain impacting more than 650,000 people across the US, Europe, and Japan. We’re 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 were also pleased to present updated nonclinical data at the ninth International Congress on Neuropathic Pain in Berlin, Germany, which demonstrated the durability, potency, and selectivity of ST-503 in nonhuman primates 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. NAV 1.7 is a well-proven target with human genetic validation, which we believe provides pain franchise potential. 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 enabling activities for the clinical trial application (CTA) for the program, ahead of our expected CTA submission as early as mid-2026.

Following on from our productive meeting with the UK’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 control (CMC) strategy for the anticipated CTA submission. In November, we presented updated preclinical data at the PRION 2025 conference, which demonstrated the 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 ST-506 in the clinic. Given the rapidly deadly nature of prion disease with no currently available treatment options, we anticipate demand for this planned study to be high with a short timeframe to an expected data readout. Furthermore, this study would mark the first in-human testing of our STAK BBB capsid, which, if successful, could unlock a broader neurology pipeline for advancement, including with potential partners. We have deliberately chosen two lead neurology assets that use different delivery mechanisms and bring different development risks. We believe both of them independently offer significant potential value and provide additional expansion opportunities into related neurological indications.

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

Prathyusha Duraibabu: 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 aftermarket offering program since September 30, will be sufficient to fund our planned operations into 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 pipeline. In the near term, we seek to bridge to that future through a balanced approach while exploring appropriate capital options. I will now hand it back to Sandy for closing remarks. Sandy?

Sandy Macrae: 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 STAR study in Fabry disease, including a positive mean annualized eGFR slope at fifty-two 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 three platform presentations on our Fabry disease program at the upcoming World Symposium in 2026. This quarter, we began recruiting and enrolling patients in the Phase 1/2 STAND study for chronic neuropathic pain, following the activation of the first two 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. Continued efforts to raise additional capital to solve our long-term funding needs remain our number one priority. As Prathyusha outlined, 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 Brumkow, Fred Ramsdale, and Simon Sakaguchi for their research on regulatory T cells or Tregs. At Sangamo, we proudly advance the clinical development of Tregs, being the first known company to dose a patient with an engineered CAR Treg. In August, we presented the clinical data from our TX 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. While we are pleased with these innovative scientific advances, we remain focused on our promising neurology pipeline. We really want to say that we are thankful to everyone who has been involved in this first-in-human study and look forward to sharing further novel scientific developments with you all. Operator, please open the line for questions.

Operator: Thank you. At this time, we will conduct a question and answer session. As a reminder, to ask a question, you will need to press 11 on your telephone and wait for your name to be announced. To withdraw your question, please press 1 again. Our next question comes from Gena Wang from Barclays. The floor is yours.

Gena Wang: Hi, thanks for taking my question. This is Han Ku 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? Also, on the regulatory front, you shared your progress with FDA. But, in light of recent news from Unicure, is there any read-through to your Fabry disease drug program?

Sandy Macrae: I can’t comment on the discussions with Unicure. 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. They confirm our ability to use eGFR data for one year to get accelerated approval. When we look at our data, the eGFR is a very clear and compelling body of data, the safety of the product, the ease of use, the cardiac data, the SF-36, the kidney benefit.

That’s what the agencies see, and that’s why they have endorsed the use of eGFR at one 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 package required for approval is essential. We are very confident in that progress. To reflect on that, know that the agency has reaffirmed our accelerated approval pathway, and this can only be helpful for our business development discussions.

Gena Wang: Thanks. How about the first part of this question? Could you share with us any progress and your status in partnership negotiation?

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

Gena Wang: Thanks a lot.

Operator: Thank you for your question. Our next question comes from Maury Raycroft from Jefferies. The floor is yours.

Maury Raycroft: Hi. Good morning, and thanks for taking my questions. Just kind of following up on the last questions. Wondering if you’re planning to have any additional meetings with FDA prior to a pre-BLA meeting. Do you need to have a pre-BLA meeting? And what additional clarification do you need from FDA at this point?

Sandy Macrae: Natalie, can you answer that?

Nathalie Dubois-Stringfellow: Yeah. 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’ll also have clarity on the elements for the CMC strategy. These interactions were recent, and we are exploring at this point if there are further topics that require a pre-BLA meeting.

Sandy Macrae: 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.

Nathalie Dubois-Stringfellow: Absolutely. And because we have RMAT, we’ve had many interactions this year, 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 the process works well, doesn’t it? Because it allows ongoing interaction.

Sandy Macrae: Exactly. Exactly.

Nathalie Dubois-Stringfellow: Yeah. We were very pleased this year with the responses from the FDA, and they were always on time, and we’ve had no delays in our discussions.

Maury Raycroft: Got it. That’s helpful. And I guess just to clarify for the BD partners, assuming that this is kind of a three-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?

Sandy Macrae: I don’t believe there are any questions left to answer. We have clarity on the primary endpoint, and the agency has seen our one-year data. That was part of the reason we had that recent clinical meeting because the last time we showed that to them, there were 18 patients at one year, and now there are 32 patients at one year and 19 patients at two years. The certainty of the eGFR slope remains, which is something that potential partners would clearly want to know. The CMC and manufacturing route is clear, and the manufacturing processes are underway and locked and loaded.

Maury Raycroft: Got it. Okay. Thanks for taking my questions.

Operator: Thank you for your question. Our next question comes from Wells Fargo. The floor is yours.

Kuan-Hung Lin: Hi. Thanks for taking our question. This is Kuan-Hung Lin for Yannan. Our question is also around the Fabry program. In your engagement with FDA, has the topic of a priority review voucher ever been mentioned? And do you think that could affect your BD discussions? Thank you.

Sandy Macrae: Natalie, can you comment on that?

Nathalie Dubois-Stringfellow: Yes. No, we have not discussed that specifically, but we’re looking into it.

Kuan-Hung Lin: Got it. Thank you. Have you shared all the details regarding the FDA meeting minutes with your potential partners, and any color you can comment on their reactions? Thank you.

Sandy Macrae: As you can imagine, when we got the minutes, we were pleased to share them with our partners or to share the essence of them. 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.

Kuan-Hung Lin: Got it. Thank you so much.

Operator: Thank you for your question. Our next question comes from Patrick Trucchio from H. C. Wainwright. The floor is yours.

Luis Santos: Good morning and thank you for taking our call. 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 ST-503 in neuropathic pain. 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 getting the study going?

Nathalie Dubois-Stringfellow: Sure. So we’re pleased to have already two sites activated that are actually screening patients right now and looking at their 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’re 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. 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 terms of the pain reduction from baseline using the PI-NRS, which is the overall pain intensity rating scale, which is broadly recognized and widely used as a preferred scale for pain measurement. We will be monitoring this in a blinded fashion throughout the dose escalation.

Sandy Macrae: Natalie, it’s been interesting the number of patients that have spontaneously written to us and tried to get into the trial. It’s clear that there’s a high unmet medical need here.

Nathalie Dubois-Stringfellow: Yeah. Absolutely. People have reached out to Sangamo or have seen the site activated on the clinicaltrials.gov page and have been directly contacting sites to see if they could be eligible throughout the country. 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 one-and-done treatment that will change their pain sensation forever. Therefore, we will understand the population better in the coming months as we dose these patients.

Luis Santos: Sounds great. Thank you for that overview. Regarding your platform, can you discuss any ongoing partner interests in the STAK BBB capsid? Any additional deals that could extend your runway? Also, with your MINT platform progressing, are there any updates?

Sandy Macrae: We’re delighted with the progress of the capsid and our department, 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, like Genentech, Astellas, and Lilly, are also pleased, and our relationship and discussions with them are going well. Greg, how’s MINT doing?

Gregory Davis: MINT is doing well, so we continue to advance that platform in ways where we have minimal spend. We’re trying to derisk that technology from a molecular standpoint for med design towards genomic targets. We’re trying to use our money wisely in that to keep that moving forward. We continue to advance business development negotiations for potential partnerships and look forward to sharing more information on those when we’re able to.

Sandy Macrae: The success of the integration with the MINT platform is increasing every time the team comes and shows me it. It really is a fundamentally important piece of science that we hope to speak about more in the future and hopefully put in the hands of others and Sangamo to make medicines from.

Luis Santos: Great. Thank you.

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

Louise Wilkie: 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.

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