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]

PART I. FINANCIAL INFORMATION [Item 1. Financial Statements](index=7&type=section&id=Item%201.%20Financial%20Statements) The company presents unaudited financial statements reporting significant net losses and substantial doubt about its ability to continue as a going concern [Condensed Consolidated Balance Sheets](index=7&type=section&id=Condensed%20Consolidated%20Balance%20Sheets) Condensed Consolidated Balance Sheet Highlights (in thousands) | Account | June 30, 2025 | December 31, 2024 | | :--- | :--- | :--- | | **Assets** | | | | Cash and cash equivalents | $38,344 | $41,918 | | Total current assets | $49,893 | $51,691 | | Total assets | $97,558 | $101,635 | | **Liabilities & Equity** | | | | Total current liabilities | $47,659 | $45,805 | | Total liabilities | $77,956 | $78,865 | | Total stockholders' equity | $19,602 | $22,770 | [Condensed Consolidated Statements of Operations](index=8&type=section&id=Condensed%20Consolidated%20Statements%20of%20Operations) Statements of Operations Highlights (in thousands, except per share amounts) | Metric | Three Months Ended June 30, 2025 | Three Months Ended June 30, 2024 | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | | :--- | :--- | :--- | :--- | :--- | | Revenues | $18,306 | $356 | $24,743 | $837 | | Total operating expenses | $36,161 | $37,440 | $72,226 | $89,447 | | Loss from operations | ($17,855) | ($37,084) | ($47,483) | ($88,610) | | Net loss | ($19,986) | ($36,128) | ($50,583) | ($85,217) | | Basic and diluted net loss per share | ($0.08) | ($0.18) | ($0.21) | ($0.44) | [Condensed Consolidated Statements of Cash Flows](index=12&type=section&id=Condensed%20Consolidated%20Statements%20of%20Cash%20Flows) Cash Flow Summary for Six Months Ended June 30 (in thousands) | Cash Flow Activity | 2025 | 2024 | | :--- | :--- | :--- | | Net cash used in operating activities | ($44,307) | ($75,547) | | Net cash (used in) provided by investing activities | ($24) | $35,967 | | Net cash provided by financing activities | $36,748 | $21,589 | | Net decrease in cash, cash equivalents, and restricted cash | ($3,574) | ($18,918) | [Notes to Condensed Consolidated Financial Statements](index=13&type=section&id=Notes%20to%20Condensed%20Consolidated%20Financial%20Statements) Notes detail accounting policies, revenue recognition, restructuring, and substantial doubt about the company's ability to continue as a going concern - Management has **substantial doubt** about the company's ability to continue as a going concern due to significant losses and limited liquidity[40](index=40&type=chunk)[41](index=41&type=chunk) - In April 2025, Sangamo entered a license agreement with Eli Lilly, receiving an **$18.0 million upfront payment** recognized as revenue in Q2 2025[83](index=83&type=chunk)[84](index=84&type=chunk)[88](index=88&type=chunk) - Pfizer terminated its collaboration agreement, resulting in a final **$5.0 million** in revenue recognized in the first half of 2025[109](index=109&type=chunk)[115](index=115&type=chunk) - The company undertook **significant restructuring**, including workforce reductions, to reduce costs and focus on neurology[140](index=140&type=chunk)[143](index=143&type=chunk) - A May 2025 underwritten offering generated aggregate net proceeds of **$21.1 million**[132](index=132&type=chunk)[133](index=133&type=chunk) [Item 2. Management's Discussion and Analysis of Financial Condition and Results of Operations](index=30&type=section&id=Item%202.%20Management's%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management discusses the company's severe liquidity crisis, recent business highlights, and the urgent need for additional capital to continue operations [Overview and Recent Business Highlights](index=30&type=section&id=Overview%20and%20Recent%20Business%20Highlights) The company reports positive clinical data and recent financing but reiterates its 'going concern' risk with cash sufficient only into Q4 2025 - The company announced **positive topline results** from the Phase 1/2 STAAR study for its Fabry disease gene therapy and plans a BLA submission[161](index=161&type=chunk) - The company is advancing its preclinical neurology programs, with the first patient in its chronic neuropathic pain study expected to be dosed in **fall 2025**[158](index=158&type=chunk)[159](index=159&type=chunk) - The company continues to seek a collaboration partner for its hemophilia A program following the termination of its agreement with Pfizer[162](index=162&type=chunk) [Results of Operations](index=34&type=section&id=Results%20of%20Operations) Financial results show a significant revenue increase from a licensing deal, while restructuring efforts have reduced overall operating expenses Revenue Comparison (in thousands) | Period | 2025 | 2024 | Change | % Change | | :--- | :--- | :--- | :--- | :--- | | Three Months Ended June 30 | $18,306 | $356 | $17,950 | 5,042.1% | | Six Months Ended June 30 | $24,743 | $837 | $23,906 | 2,856.0% | - The revenue increase was primarily due to an **$18.0 million** upfront payment from the Lilly agreement and a **$5.0 million** payment from the terminated Pfizer collaboration[175](index=175&type=chunk)[176](index=176&type=chunk) Operating Expense Comparison (in thousands) | Expense Category | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | Change | % Change | | :--- | :--- | :--- | :--- | :--- | | Research and development | $53,090 | $60,114 | ($7,024) | (12%) | | General and administrative | $19,136 | $23,812 | ($4,676) | (20%) | | Impairment of long-lived assets | $0 | $5,521 | ($5,521) | (100%) | | **Total operating expenses** | **$72,226** | **$89,447** | **($17,221)** | **(19%)** | [Liquidity and Capital Resources](index=36&type=section&id=Liquidity%20and%20Capital%20Resources) The company details its severe liquidity constraints, with cash reserves sufficient only into Q4 2025, raising substantial doubt about its going concern status - As of June 30, 2025, the company had cash and cash equivalents of **$38.3 million**, down from $41.9 million at year-end 2024[193](index=193&type=chunk) - The company raised approximately **$21.1 million** from an underwritten offering and **$17.6 million** from its ATM program in the first half of 2025[194](index=194&type=chunk)[150](index=150&type=chunk) - Management concludes there is **substantial doubt** about the company's ability to continue as a going concern, as existing cash funds operations only into Q4 2025[197](index=197&type=chunk) [Item 3. Quantitative and Qualitative Disclosures about Market Risk](index=39&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20about%20Market%20Risk) As a smaller reporting company, this disclosure is not required [Item 4. Controls and Procedures](index=39&type=section&id=Item%204.%20Controls%20and%20Procedures) Management concluded that disclosure controls and procedures were effective, with no material changes to internal controls during the quarter PART II. OTHER INFORMATION [Item 1. Legal Proceedings](index=41&type=section&id=Item%201.%20Legal%20Proceedings) The company is not currently a party to any material pending legal proceedings [Item 1A. Risk Factors](index=41&type=section&id=Item%201A.%20Risk%20Factors) Key risks include substantial doubt about going concern, potential Nasdaq delisting, and regulatory or trade policy disruptions - The company's financial position raises **substantial doubt** about its ability to continue as a going concern, risking cessation of operations or bankruptcy[218](index=218&type=chunk)[219](index=219&type=chunk) - Sangamo received a **Nasdaq deficiency notice** for its stock price falling below the $1.00 minimum bid requirement and faces potential delisting[225](index=225&type=chunk) - A new risk factor addresses potential **disruptions at the FDA**, which could adversely impact the Accelerated Approval pathway for its product candidates[234](index=234&type=chunk)[235](index=235&type=chunk) - The company identifies risks related to **international trade policies**, which could increase expenses and disrupt its supply chain[230](index=230&type=chunk)[231](index=231&type=chunk) [Items 2-6. Other Required Information](index=44&type=section&id=Items%202-6.%20Other%20Required%20Information) This section confirms no unregistered equity sales, defaults, or other material information, and lists the exhibits filed with the report

[Sangamo Therapeutics Q2 2025 Earnings Release](index=1&type=section&id=Sangamo%20Therapeutics%20Q2%202025%20Earnings%20Release) Sangamo Therapeutics reports significant Q2 2025 progress in its Fabry disease and neurology pipelines, alongside improved financial results driven by increased revenues and reduced net loss [Business Highlights](index=1&type=section&id=Recent%20Business%20Highlights) Sangamo Therapeutics announced significant progress across its pipeline in Q2 2025, including positive Fabry disease study results, new clinical site initiation for neuropathic pain, and productive discussions for its prion disease program, alongside a $21 million equity offering [Corporate Updates](index=1&type=section&id=Corporate%20Updates) The company successfully raised approximately $21 million in net proceeds from a recent equity offering - Raised approximately **$21 million** in net proceeds from an underwritten registered equity offering[5](index=5&type=chunk) [Fabry Disease Program (isaralgagene civaparvovec / ST-920)](index=1&type=section&id=Fabry%20Disease) Positive topline results from the STAAR study for Fabry disease gene therapy support an accelerated approval pathway with the FDA - Announced positive topline results from the registrational Phase 1/2 STAAR study for isaralgagene civaparvovec (ST-920) in adults with Fabry disease[1](index=1&type=chunk)[6](index=6&type=chunk) Key Efficacy Endpoints from STAAR Study | Endpoint | Result | Follow-up Period | Patient Count | | :--- | :--- | :--- | :--- | | **Mean Annualized eGFR Slope** | +1.965 mL/min/1.73m²/year | 52 weeks | 32 | | **Mean Annualized eGFR Slope** | +1.747 mL/min/1.73m²/year | 104 weeks | 19 | | **α-Gal A Activity** | Maintained elevated expression | Up to 4.5 years | Longest treated patient | | **Plasma lyso-Gb3 Levels** | Remained generally stable | Post-ERT withdrawal | N/A | - The FDA has agreed that the positive mean annualized eGFR slope at 52 weeks will serve as the primary basis for a Biologics License Application (BLA) submission under the Accelerated Approval pathway, anticipated as early as Q1 2026[1](index=1&type=chunk)[6](index=6&type=chunk)[11](index=11&type=chunk) - The treatment demonstrated a favorable safety and tolerability profile without the need for preconditioning, with most adverse events being grade 1-2[11](index=11&type=chunk) [Core Neurology Pipeline](index=2&type=section&id=Core%20Neurology%20Pipeline) Sangamo initiated its first clinical site for chronic neuropathic pain and aligned on study design for its prion disease program - **Chronic Neuropathic Pain (ST-503):** The first clinical site has been initiated for the Phase 1/2 STAND study, with the first patient expected to be dosed in fall 2025 and preliminary efficacy data anticipated in Q4 2026[2](index=2&type=chunk)[11](index=11&type=chunk) - **Prion Disease (ST-506):** Held a productive meeting with the UK's MHRA, aligning on study design, with a Clinical Trial Application (CTA) submission expected as early as mid-2026[2](index=2&type=chunk)[11](index=11&type=chunk) [Second Quarter 2025 Financial Results](index=2&type=section&id=Second%20Quarter%202025%20Financial%20Results) For Q2 2025, Sangamo reported significantly increased revenues to $18.3 million and a narrowed net loss, with cash and equivalents of $38.3 million expected to fund operations into Q4 2025 [Key Financial Metrics](index=2&type=section&id=Key%20Financial%20Metrics) Sangamo's Q2 2025 revenues significantly increased to $18.3 million, leading to a reduced net loss of $20.0 million Q2 2025 vs. Q2 2024 Performance | Metric | Q2 2025 | Q2 2024 | | :--- | :--- | :--- | | **Revenues** | $18.3 million | $0.3 million | | **Net Loss** | ($20.0 million) | ($36.1 million) | | **Net Loss per Share** | ($0.08) | ($0.18) | - The **$18.0 million** increase in revenues was primarily attributable to an upfront license payment received under a capsid license agreement with Eli Lilly and Company[10](index=10&type=chunk) [Operating Expenses](index=3&type=section&id=GAAP%20and%20Non-GAAP%20Operating%20Expenses) GAAP operating expenses decreased in Q2 2025 due to strategic realignment, while non-GAAP expenses slightly increased Operating Expenses for Q2 (in millions) | Expense Type | Q2 2025 | Q2 2024 | | :--- | :--- | :--- | | **GAAP Operating Expenses** | $36.2 | $37.4 | | **Non-GAAP Operating Expenses** | $33.0 | $31.9 | - The decrease in GAAP operating expenses was mainly due to cost reductions from strategic realignment, including lower headcount and facilities costs, partially offset by increased expenses for BLA readiness activities for the Fabry disease program[13](index=13&type=chunk) [Cash Position and Financial Guidance](index=3&type=section&id=Cash%20and%20Financial%20Guidance) Sangamo's cash and equivalents of $38.3 million are projected to fund operations into Q4 2025, with full-year 2025 operating expense guidance reiterated - Cash and cash equivalents were **$38.3 million** as of June 30, 2025, sufficient to fund planned operations into the fourth quarter of 2025 with recent at-the-market offering proceeds[14](index=14&type=chunk) Reiterated Full-Year 2025 Financial Guidance | Metric | Expected Range (in millions) | | :--- | :--- | | **GAAP Total Operating Expenses** | $135 - $155 | | **Non-GAAP Total Operating Expenses** | $125 - $145 | [Corporate Information and Events](index=3&type=section&id=Corporate%20Information%20and%20Events) Sangamo Therapeutics will participate in two investor conferences in September 2025 and held a conference call on August 7, 2025, to provide updates - Sangamo plans to participate in the Cantor Global Healthcare Conference and the Wells Fargo Healthcare Conference from September 3-5, 2025[17](index=17&type=chunk)[19](index=19&type=chunk) - The management team held a conference call on August 7, 2025, to discuss program and financial updates[18](index=18&type=chunk) [Financial Statements](index=6&type=section&id=SELECTED%20CONSOLIDATED%20FINANCIAL%20DATA) The consolidated financial statements detail Sangamo's Q2 2025 performance, showing increased revenues, reduced net loss, and a cash position of $38.3 million [Statement of Operations](index=6&type=section&id=Statement%20of%20Operations%20Data) The Statement of Operations shows a significant increase in revenues and a reduction in net loss for both the three and six months ended June 30, 2025 Consolidated Statement of Operations Data (Unaudited, in thousands) | | Three Months Ended June 30, | Six Months Ended June 30, | | :--- | :--- | :--- | :--- | :--- | | | **2025** | **2024** | **2025** | **2024** | | **Revenues** | $18,306 | $356 | $24,743 | $837 | | **Total operating expenses** | $36,161 | $37,440 | $72,226 | $89,447 | | **Loss from operations** | ($17,855) | ($37,084) | ($47,483) | ($88,610) | | **Net loss** | ($19,986) | ($36,128) | ($50,583) | ($85,217) | | **Net loss per share** | ($0.08) | ($0.18) | ($0.21) | ($0.44) | [Balance Sheet](index=6&type=section&id=Selected%20Balance%20Sheet%20Data) The Balance Sheet indicates total assets of $97.6 million and cash and cash equivalents of $38.3 million as of June 30, 2025 Selected Balance Sheet Data (Unaudited, in thousands) | | June 30, 2025 | December 31, 2024 | | :--- | :--- | :--- | | **Cash and cash equivalents** | $38,344 | $41,918 | | **Total assets** | $97,558 | $101,635 | | **Total stockholders' equity** | $19,602 | $22,770 | [Disclosures](index=4&type=section&id=Disclosures) This section outlines forward-looking statements regarding financial risks and clinical uncertainties, and explains the rationale for using non-GAAP financial measures - **Forward-Looking Statements:** Key risks highlighted include the company's lack of capital resources, the need for substantial additional funding to continue as a going concern, the uncertain R&D process, and the unpredictable regulatory approval process[23](index=23&type=chunk) - **Non-GAAP Financial Measures:** The company presents non-GAAP operating expenses, which exclude items like depreciation, stock-based compensation, and impairment charges, to provide what it believes is a more meaningful comparison of its operational performance over time[25](index=25&type=chunk)

Core Points - Sangamo Therapeutics, Inc. is set to release its second quarter 2025 financial results on August 7, 2025, after market close [1] - A conference call will be held at 4:30 p.m. Eastern on the same day to discuss financial results and provide business updates [1] - Participants can register for the call via a provided link and are encouraged to join 10 minutes early [2] Company Overview - Sangamo Therapeutics focuses on genomic medicine, aiming to develop treatments for serious neurological diseases lacking adequate 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]

Financial Data and Key Metrics Changes - The company reported a significant reduction in non-GAAP operating expenses by 50% year-on-year in 2024, focusing on key priorities to enhance efficiency [17] - An equity offering was announced to extend the company's cash runway, with proceeds expected to last until late Q3 2025 [16][12] Business Line Data and Key Metrics Changes - The capsid engineering platform saw a new licensing agreement with Eli Lilly, which includes an $18 million upfront fee and potential total payments of up to $1.4 billion across five targets [6][7] - The neurology pipeline is advancing, with preparations for a Phase 1/2 study of ST503 for chronic neuropathic pain expected to begin in mid-2025 [8][10] Market Data and Key Metrics Changes - The company is actively engaging in discussions with potential partners for its Fabry disease program, with a focus on securing a commercial partnership that aligns with its neurology mission [11][19] Company Strategy and Development Direction - The long-term vision is to position Sangamo as a neurology-focused genomic medicine company, aiming to deliver transformational therapies and significant shareholder value [15] - The company is committed to operating efficiently while advancing its neurology programs, with a disciplined approach to capital allocation [17] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism regarding the FDA's engagement and the clarity provided during the Type B meeting, which is crucial for the BLA submission process [32] - The company remains focused on securing funding and partnerships to support its neurology pipeline and Fabry disease program [19] Other Important Information - The company plans to present data at the ASGCT Annual Meeting, showcasing advancements in its neurology pipeline and the effectiveness of its technologies [19][9] Q&A Session Summary Question: Can you provide more color on the top line eGFR data? - Management confirmed that the updated mean eGFR slope will be shared, along with additional information at a later date [24] Question: How many potential partners are you currently in conversations with for STABBI? - Management indicated that multiple potential partners are being engaged, and the Type B meeting was beneficial for discussions [27] Question: Has the pace of conversations with potential partners changed due to the macro landscape? - Management noted that interactions with the FDA have been positive and have not impacted the program [33] Question: Will you file based on 52-week eGFR data? - Management confirmed that they are pursuing the agreement with the FDA to use 52-week eGFR data for the entire patient population [38] Question: Are all patients who were on ERT still off ERT? - Management confirmed that all 18 patients who started on ERT are still off ERT [47]

Financial Data and Key Metrics Changes - The company reported a significant reduction in non-GAAP operating expenses by 50% year-on-year in 2024, focusing on key priorities to enhance efficiency [16][11] - An equity offering was announced to extend the company's cash runway, which is expected to last until late in the third quarter of 2025 [15][11] Business Line Data and Key Metrics Changes - The company signed a third capsid license agreement with Eli Lilly, receiving an upfront fee of $18 million and potential additional payments of up to $1.4 billion across five disease targets [5][6] - The Fabry program achieved key de-risking milestones, with all patients in the Phase 1/2 STAR study completing at least 52 weeks of follow-up, indicating a positive mean eGFR slope [9][10] Market Data and Key Metrics Changes - The company is actively engaging in discussions with multiple potential partners for its Fabry disease program, indicating strong interest in its technology [12][32] - The FDA's recent interactions have been positive, with no significant changes in their approach to the company's programs, including Fabry disease [32][33] Company Strategy and Development Direction - The long-term vision is to focus on neurology as a core area, aiming to deliver transformational therapies while maximizing shareholder value [13][15] - The company is committed to securing a commercial partnership for the Fabry program to support its neurology mission and ensure sustainable funding [11][18] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the FDA's engagement and the clarity provided regarding the chemistry, manufacturing, and controls pathway for the BLA submission [10][32] - The company remains focused on advancing its neurology pipeline and securing necessary funding to support ongoing clinical trials [11][18] Other Important Information - The company plans to present data at the ASGCT Annual Meeting, showcasing advancements in its neurology pipeline and the effectiveness of its capsid delivery technology [8][18] - The company is exploring additional funding opportunities through collaborations related to its technology platforms [12] Q&A Session Summary Question: Can you provide more color on what exactly you plan to show in the top line eGFR data? - Management confirmed that they will share the updated mean eGFR slope and additional information at a later date [21][23] Question: How many potential partners are you currently in conversations with regarding STABBI? - Management indicated that there are multiple potential partners but did not disclose specific details [26] Question: Has the pace of conversations with potential partners changed due to the macro landscape? - Management reported that interactions with the FDA have been positive and have not impacted their program [33] Question: Do you still plan to file based on 52-week eGFR data? - Management confirmed that they are pursuing the agreement with the FDA to use 52-week eGFR data for the entire patient population [37] Question: Are all patients who were on ERT still off ERT? - Management confirmed that all 18 patients who started on ERT are still off ERT [46] Question: Is there any threshold for the eGFR slope that should be looked at? - Management stated they are not commenting on specific statistical significance at this time but confirmed the mean eGFR slope remains positive [48][49]

Core Viewpoint - Sangamo Therapeutics reported a quarterly loss of $0.14 per share, which was worse than the Zacks Consensus Estimate of a loss of $0.11, marking an earnings surprise of -27.27% [1] - The company generated revenues of $6.44 million for the quarter, missing the consensus estimate by 29.77%, compared to $0.48 million in the same quarter last year [2] Group 1: Financial Performance - The company has surpassed consensus EPS estimates only once in the last four quarters [2] - Sangamo shares have declined approximately 31.4% year-to-date, contrasting with the S&P 500's decline of -3.8% [3] Group 2: Future Outlook - The current consensus EPS estimate for the upcoming quarter is -$0.09 on revenues of $14.8 million, and for the current fiscal year, it is -$0.39 on revenues of $46.97 million [7] - The estimate revisions trend for Sangamo is currently unfavorable, resulting in a Zacks Rank 4 (Sell), indicating expected underperformance in the near future [6] Group 3: Industry Context - The Medical - Biomedical and Genetics industry is ranked in the top 34% of over 250 Zacks industries, suggesting a favorable outlook compared to lower-ranked industries [8] - Empirical research indicates a strong correlation between near-term stock movements and trends in earnings estimate revisions, which can be tracked by investors [5]

Delivering the Future of Genomic Medicines May 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 dise ...