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]

Neurology Pipeline and Technology - Sangamo's STAC-BBB capsid demonstrates industry-leading performance with 700-fold higher transgene expression than benchmark capsid AAV9[25] - ST-503 for chronic neuropathic pain is advancing with patient enrollment and dosing expected in mid-2025 and preliminary proof of efficacy data anticipated in Q4 2026[16, 35, 39] - Prion disease program is progressing with a CTA submission anticipated in Q1 2026 and preliminary clinical data expected in Q4 2026[12, 20, 35, 39] - ST-503 resulted in approximately 70% repression of the SCN9A gene in mice, reducing pain hypersensitivity[18] - Intrathecal delivery of ZFR in NHPs by AAV9 demonstrated up to 60% repression of SCN9A in dorsal root ganglia (DRG) tissue[18] Partnerships and Financials - Sangamo received an $18 million upfront license fee from Lilly for a capsid license agreement to deliver genomic medicines for up to five diseases of the CNS, with potential for up to $1.4 billion in additional licensed target fees and milestone payments, plus tiered royalties on potential net sales[30, 36] - STAC-BBB partnerships have the potential for up to $4.6 billion in future milestones and exercise fees, assuming exercise of all options and targets, plus additional potential product royalties[26] - As of March 31, 2025, Sangamo had approximately $25.2 million in cash and cash equivalents[36] Fabry Disease Program - A potential BLA submission for Isaralgagene civaparvovec is anticipated as early as the first quarter of 2026[13, 28, 35, 39] - All dosed patients (32) in Phase 1/2 STAAR study data have now completed at least 52-weeks of follow-up, with preliminary analysis indicating a positive mean eGFR slope[35, 39, 130] - In the Phase 1/2 STAAR study, a positive mean eGFR slope of 3.061 mL/min/1.73m2/year (95% confidence interval: 0.863, 5.258) was observed in the 23 patients that have reached 1-year follow-up[136]

Financial Data and Key Metrics Changes - The company reported revenue of $47 million for Q1 2025, exceeding expectations and showing growth of over $4 million from Q4 2024 [7][30] - GAAP net loss before noncontrolling interests was $53 million for Q1 2025, compared to a loss of $23 million in Q1 2024 [30] - Adjusted EBITDA was a negative $11 million for Q1 2025, down from a positive $8 million in Q1 2024 [30][32] - Basic and diluted EPS for Q1 was a loss of $0.21 per share, compared to a loss of $0.09 per share in Q1 2024 [33] Business Line Data and Key Metrics Changes - The Nucleic Acid Production (NAP) segment generated $29 million in revenue for Q1 2025, up $1 million from Q4 2024 [7] - The Biologics Safety Testing (BST) segment reported revenue of $18 million in Q1 2025, an increase of $3 million over Q4 2024 [7][39] - The adjusted EBITDA margin for the BST segment was 70%, indicating strong performance [39] Market Data and Key Metrics Changes - Revenue by customer type in Q1 was 29% from biopharma, 28% from life science and diagnostics, 6% from academia, 7% from CDMO, and 30% through distributors [8] - Revenue by geography was 62% from North America, 15% from EMEA, 15% from Asia Pacific, and 8% from China [8] Company Strategy and Development Direction - The company is focused on a return to growth strategy and building a diversified, predictable franchise as a life science tool provider [11] - The strategy includes vertical integration in the NAP segment to enhance quality, speed, and cost efficiency [9] - The company aims to advance its market leadership in genomic medicines and expand its product and service portfolio [11][20] Management's Comments on Operating Environment and Future Outlook - Management acknowledged the challenges of transitioning from a COVID-driven revenue model to a more diversified business [43] - The company is actively monitoring trade dynamics and working to mitigate potential impacts from tariffs [10][44] - There is a strong cash position of $285 million, which is seen as sufficient to manage the current reset period [44] Other Important Information - The company plans to publish its 2024 sustainability report, highlighting its commitment to sustainability and long-term strategic objectives [27][28] - The integration of recent acquisitions is progressing well, enhancing operational capabilities and supply chain control [35] Q&A Session Summary Question: What does the intel tell about the focus areas for new trials and those being discontinued? - Management noted that while preclinical programs were flat, clinical programs showed growth, indicating a focus on later-stage projects due to funding conservatism [50][51] Question: What are the biggest drivers of incremental demand in the next 12-18 months? - Management indicated that clinical progression and new business wins would drive demand, with a focus on expanding from bulk reagent supply to comprehensive service offerings [52][54] Question: Is there an opportunity to take market share with 100% U.S. manufacturing? - Management sees renewed interest in their U.S.-based manufacturing capabilities, which could attract customers looking for alternative suppliers [57][58] Question: What are the buying patterns from biopharma and academic customers? - The response highlighted a mixed environment, with some customers securing long-term funding while others are delaying decisions due to funding uncertainties [63][64] Question: Is there any government work or exposure to BARDA? - Management confirmed that there is no direct government work currently, and past BARDA engagements have been satisfied [69][70] Question: Is there seasonality in BST revenue? - Management indicated that Q1 is typically a high point for BST revenue due to manufacturing cycles, and a seasonal dip is expected in subsequent quarters [74][76]