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]

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 ...

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]