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]

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]