Key clinical assets include ST-920 (Phase 1/2 for Fabry disease), TX200 (Phase 1/2 for kidney transplant rejection), and SB-525 (Phase 3 for hemophilia A). The company maintains significant strategic partnerships with Biogen, Pfizer, Sanofi, Novartis, and Kite Pharma, with preclinical programs targeting neurological diseases including ALS/FTD and Huntington's.
Cyborg Score Rationale
2024 revenue was $57.8M (down 67.2% year-over-year) with a market cap of $138.2M as of that period. The company is pre-commercial with significant cash burn and reliance on partnerships and capital raises. Cash position of $38.3M as of June 2025 was expected to fund operations into Q4 2025, creating near-term liquidity concerns.
Top Insights
FDA acceptance of rolling BLA for ST-920 (Fabry gene therapy) in November 2025 offers clearest path to commercial product using accelerated approval pathway
Launched Phase 1/2 chronic pain trial (STAND study) with ST-503 in late 2025, marking clinical debut of epigenetic regulation technology
Secured $18M non-dilutive funding from Lilly in Q1 2025 for capsid license agreement, validating proprietary delivery platform technology
Company is actively fundraising to extend cash runway; early February 2026 underwritten offering announced to support operations into 2026
Named Competitors
Gene Therapy Platform — CRISPR gene editing for genetic diseases
Gene Therapy Platform — AAV-based gene therapy for hemophilia and other genetic disorders
Cell Therapy Platform — CRISPR-based in vivo gene editing
Recent Developments
(February 2026) Announced underwritten offering for 35.2M shares to address liquidity needs
(December 2025) FDA grants Fast Track Designation to ST-503 for chronic neuropathic pain treatment
(November 2025) FDA accepts rolling BLA for ST-920 Fabry disease gene therapy with accelerated approval potential
(Q1 2025) Closed $18M non-dilutive licensing deal with Lilly for capsid platform technology
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