Delivering the Future of Genomic Medicines November 2025 Forward-Looking Statements and Legal Disclaimers This presentation, and accompanying oral commentary, contains forward-looking statements regarding our current expectations. These forward-looking statements include, without limitation, statements relating to: the therapeutic and commercial potential and value of our product candidates and engineered capsids, including the ability of our zinc finger epigenetic regulators to address various neurological ...

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 ________________________________________________ FORM 10-Q ________________________________________________ (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended September 30, 2025 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission file number 000-30171 __ ...

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Held productive interaction with Medicines and Healthcare products Regulatory Agency (MHRA) to discuss prion disease study ahead of anticipated Clinical Trial Application (CTA) submission. RICHMOND, California, November 6, 2025 - Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, today reported recent business highlights and third quarter 2025 financial results. "We continued to advance our clinical and pre-clinical pipeline this quarter and are excited to now be recruiting and enrolling ...

Held meeting with U.S. Food and Drug Administration (FDA) where, among other things, they reiterated October 2024 agreement to use estimated glomerular filtration rate (eGFR) slope as an endpoint to support accelerated approval pathway for isaralgagene civaparvovec in Fabry disease. Presented detailed clinical data from registrational STAAR study showing potential for isaralgagene civaparvovec as a one-time, durable treatment of underlying pathology of Fabry disease to provide meaningful, multi-organ, clini ...

Core Insights - Sangamo Therapeutics, Inc. is set to release its third quarter 2025 financial results on November 6, 2025, before market opening [1] - A conference call will be held at 8:30 a.m. Eastern on the same day to discuss financial results and provide business updates [1] Company Overview - Sangamo Therapeutics is focused on genomic medicine, aiming to develop treatments for serious neurological diseases lacking adequate treatment options [4] - The company utilizes zinc finger epigenetic regulators and a capsid discovery platform to potentially address neurological disorders and expand delivery methods [4] - Sangamo's pipeline includes multiple partnered programs and opportunities for further partnerships and investments [4]

Core Insights - The data supports the potential of isaralgagene civaparvovec as a one-time, durable treatment for Fabry disease, offering significant multi-organ clinical benefits compared to current care standards [1][6] - The STAAR study showed a positive mean annualized estimated glomerular filtration rate (eGFR) slope at 52 weeks across all patients, which the FDA has agreed will be the primary basis for approval [1][6] - Sangamo plans to submit a Biologics License Application (BLA) in 2026 under the Accelerated Approval pathway [1][6] Efficacy - The STAAR study included 32 dosed patients with a positive mean annualized eGFR slope of 1.965 mL/min/1.73m/year at 52 weeks, which is favorable compared to approved Fabry treatments [4] - At Week 104, a mean annualized eGFR slope of 1.747 mL/min/1.73m/year was observed for 19 patients [4] - Stable cardiac function was maintained over at least one year, with various cardiac metrics remaining stable [4] Safety - Isaralgagene civaparvovec demonstrated a favorable safety and tolerability profile, with most adverse events being grade 1-2 [12] - The most common treatment-emergent adverse events included pyrexia (60.6%), COVID-19 (36.4%), headache (30.3%), and nasopharyngitis (33.3%) [12] - All treatment-emergent adverse events resolved with clinical management, and there were no safety-related study discontinuations or deaths [12] Regulatory Designations - Isaralgagene civaparvovec has received Orphan Drug, Fast Track, and RMAT designations from the FDA, as well as Orphan Medicinal Product designation from the European Medicines Agency [8] - The company is preparing for BLA submission while engaging in business development for potential commercialization [8] Study Design - The Phase 1/2 STAAR study is a global open-label, single-dose, dose-ranging, multicenter clinical study designed to evaluate isaralgagene civaparvovec in Fabry disease patients [10] - The study enrolled male and female patients, with a median age of 42 and a median follow-up duration of 24 months [7] Quality of Life Improvements - Statistically significant improvements in quality of life scores were observed, including role-physical (+14.8), vitality (+9.6), and bodily pain (+9.0) at week 52 compared to baseline [12] - Improvements in disease severity were reported in the Mainz Severity Score Index, with 22 patients showing total score improvements at 12 months [12]

Core Viewpoint - Sangamo Therapeutics (SGMO) has received an upgrade to a Zacks Rank 2 (Buy), indicating a positive outlook based on rising earnings estimates, which are a significant factor influencing stock prices [1][3]. Earnings Estimates and Stock Performance - The Zacks rating system emphasizes the correlation between changes in earnings estimates and stock price movements, suggesting that revisions in earnings estimates can lead to significant near-term price changes [4][6]. - For Sangamo, the Zacks Consensus Estimate has increased by 10% over the past three months, reflecting a positive trend in earnings expectations [8]. Zacks Rating System - The Zacks Rank system categorizes stocks into five groups based on earnings estimates, with a strong historical performance, particularly for Zacks Rank 1 stocks, which have averaged a +25% annual return since 1988 [7]. - Sangamo's upgrade to Zacks Rank 2 places it in the top 20% of Zacks-covered stocks, indicating a strong potential for market-beating returns in the near term [10].

Financial Data and Key Metrics Changes - The company reported positive top-line results from the registrational STAR study in Fabry disease, with a mean annualized estimated glomerular filtration rate (eGFR) slope of almost 2 observed at 52 weeks across all 32 patients [7][8] - The FDA has agreed that the mean eGFR slope will serve as the primary basis for approval under the accelerated approval pathway [8][14] - The company completed an equity offering to bridge to an anticipated Fabry commercialization agreement, with the current cash runway expected to fund operations into 2025 [20] Business Line Data and Key Metrics Changes - The Fabry disease program showed a positive annualized eGFR slope of 1.7 for 19 patients who achieved two years of follow-up, compared to an average untreated decline of -3 to -4 [9][12] - The neurology pipeline program initiated its first clinical site for the Phase one/two STAND study in chronic neuropathic pain, with plans to dose the first patient in fall 2025 [15][19] Market Data and Key Metrics Changes - The company is engaging in business development negotiations for a potential Fabry commercialization agreement and broader discussions across its pipeline and platforms [20] - There is strong enthusiasm from both patients and physicians regarding the potential adoption of the Fabry treatment, with patients expressing a desire for better solutions compared to current standard care [40][41] Company Strategy and Development Direction - The company aims to secure a commercialization partner for its Fabry treatment while advancing its neurology genomic medicine pipeline [20] - The strategic focus includes addressing long-term funding needs to support the promising neurology pipeline and ensuring successful clinical trial outcomes [20] Management's Comments on Operating Environment and Future Outlook - Management expressed pride in the progress made despite a challenging environment, highlighting the importance of the recent clinical advancements [6][19] - The company anticipates preliminary proof of efficacy data for the STAND study in 2026 and is preparing for a BLA submission for the Fabry treatment as early as Q1 2026 [14][19] Other Important Information - The company held a productive meeting with the UK's MHRA regarding the prion disease program, aligning on planned studies and expected CTA submission by mid-2026 [16][17] - The company showcased its epigenetic regulation and capsid delivery technology at a recent conference, emphasizing the potential of its prion disease treatment [17] Q&A Session Summary Question: Has the team held the pre-BLA meeting with the FDA regarding the one-year eGFR data? - The company has not yet held the pre-BLA meeting but plans to do so in the future, with the FDA previously agreeing that one-year eGFR data could suffice for accelerated approval [24][25] Question: What additional insights should be anticipated at the upcoming presentation? - The company plans to present top-line data with additional details compared to previous releases, but individual patient data will not be shown [26][27] Question: How does the efficacy of ST-503 compare to recent small molecule Nav1.8 inhibitors? - Management remains convinced that targeting NaV1.7 is the right approach, citing evidence of its fundamental role in pain signaling [32][34] Question: Have any surveys been conducted to understand potential adoption rates for the Fabry treatment? - Feedback from patient advocacy groups indicates a strong desire for the treatment, with patients eager for a one-time injection solution compared to the burdensome current standard of care [39][40] Question: What is the status of discussions with potential partners? - All potential partners have expressed excitement about the data and are reassured by the company's interactions with the FDA, which have de-risked the product [43][45]

Neurology Focus and Technology - Sangamo is focused on developing genomic medicines for debilitating neurological diseases[3, 135] - The company utilizes potent zinc finger epigenetic regulation technology and an industry-leading AAV capsid discovery platform for brain delivery[4, 136] - STAC-BBB capsid has demonstrated robust penetration of the blood-brain barrier and widespread transgene expression throughout the brain in NHPs, showing up to 700-fold higher transgene expression than benchmark capsid AAV9[22, 24, 98] Pipeline and Milestones - Phase 1/2 STAND study of ST-503 for idiopathic small fiber neuropathy (iSFN) has initiated, with patient dosing expected in Fall 2025 and preliminary proof of efficacy data anticipated in Q4 2026[12, 16, 33, 38] - CTA submission for ST-506 in prion disease is anticipated as early as mid-2026, with clinical trial enrollment and dosing expected in late-2026 and preliminary clinical data in mid-2027[12, 19, 33, 38, 94] Fabry Disease Program - Positive topline results from the registrational STAAR study in Fabry disease showed a positive mean annualized eGFR slope of 1.965 mL/min/1.73m2/year at 52 weeks across all 32 dosed patients[12, 29, 33, 38, 131] - A BLA submission for isaralgagene civaparvovec is expected as early as Q1 2026, with ongoing business development negotiations for a potential commercialization agreement[12, 27, 33, 38, 130, 131] Financial Status - The company had approximately $38.3 million in cash and cash equivalents as of June 30, 2025[34, 40] - Approximately $21 million in net proceeds were raised from an underwritten registered equity offering[34, 38] - Cash received from partners to date is $88 million[25] - Up to $4.6 billion in potential future milestones and exercise fees assuming exercise of all options and targets[25]