Sangamo Therapeutics(US:SGMO)2025-05-12 22:52Neurology 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]