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]