Company Description
Sangamo Therapeutics, Inc. (Nasdaq: SGMO) is a genomic medicine company focused on developing medicines for patients and families affected by serious neurological diseases and other conditions with limited or no treatment options. According to multiple company disclosures, Sangamo is advancing a neurology-focused pipeline that applies its zinc finger epigenetic regulation technology, gene therapy platforms and capsid discovery capabilities to address devastating disorders, including Fabry disease, chronic neuropathic pain and prion disease.
The company describes itself as dedicated to translating ground‑breaking science into medicines that can transform patients’ lives. Sangamo believes its zinc finger epigenetic regulators are well suited to potentially address severe neurological disorders, and that its proprietary capsid discovery platform, including the STAC‑BBB and SIFTER technologies, can expand delivery of genomic medicines beyond currently available intrathecal delivery capsids, particularly to the central nervous system. Sangamo’s pipeline also includes multiple partnered programs and programs with opportunities for partnership and investment.
Fabry disease program: isaralgagene civaparvovec (ST‑920)
A central element of Sangamo’s business is its wholly owned investigational gene therapy isaralgagene civaparvovec, also known as ST‑920, for adults with Fabry disease. Fabry disease is described in Sangamo’s communications as a lysosomal storage disorder caused by mutations in the galactosidase alpha (GLA) gene, which lead to deficient alpha‑galactosidase A (α‑Gal A) enzyme activity and accumulation of globotriaosylceramide (Gb3). This accumulation can damage vital organs, including the kidney, heart, nerves, eyes, gut and skin, and is associated with symptoms such as neuropathic pain, gastrointestinal disturbance, kidney disease, heart failure and other systemic complications.
ST‑920 is being evaluated in the registrational Phase 1/2 STAAR study, a global open‑label, single‑dose, dose‑ranging, multicenter clinical study in Fabry patients. Company reports state that a single intravenous infusion of isaralgagene civaparvovec, without preconditioning, led to a positive mean annualized estimated glomerular filtration rate (eGFR) slope at 52 weeks across all dosed patients, which the U.S. Food and Drug Administration (FDA) has agreed can serve as an endpoint to support an accelerated approval pathway. Longer‑term follow‑up has shown positive mean annualized eGFR slopes at 104 weeks in patients with two years of data.
Sangamo has reported that ST‑920 treatment was associated with sustained α‑Gal A activity, stability of plasma lyso‑Gb3 levels, stable cardiac function measures, and improvements in Fabry disease severity scores, quality of life assessments and gastrointestinal symptom scales in the STAAR study. All patients who entered the study on enzyme replacement therapy (ERT) were able to withdraw from ERT under medical supervision, with plasma biomarkers generally remaining stable as of specified data cutoffs. Across company communications, isaralgagene civaparvovec is described as demonstrating a favorable safety and tolerability profile, with most adverse events graded as mild or moderate and no safety‑related study discontinuations reported in the summarized data.
Regulatory disclosures note that isaralgagene civaparvovec has received Orphan Drug, Fast Track and RMAT designations from the FDA, as well as Orphan Medicinal Product designation and PRIME eligibility from the European Medicines Agency and participation in the Innovative Licensing and Access Pathway from the U.K. Medicines and Healthcare products Regulatory Agency. Sangamo has stated that it is preparing a Biologics License Application (BLA) for ST‑920 under the FDA’s accelerated approval program and has initiated a rolling BLA submission process, with the FDA accepting a request for rolling review.
Neurology pipeline: chronic neuropathic pain and prion disease
Beyond Fabry disease, Sangamo highlights a core neurology pipeline built around epigenetic regulation and neurotropic capsid delivery. The company is advancing ST‑503, an investigational epigenetic regulator for intractable pain due to small fiber neuropathy (SFN), a form of chronic neuropathic pain. ST‑503 is being studied in the Phase 1/2 STAND study, a multicenter, double‑blind, randomized, sham‑controlled, dose‑escalation trial assessing the safety, tolerability and preliminary efficacy of a one‑time intrathecal dose in adults with SFN whose pain has been refractory to first‑line medical therapies. Sangamo has reported that the FDA granted Fast Track Designation to ST‑503 for the treatment of intractable pain due to SFN, and that clinical site activation, patient recruitment and enrollment activities are underway.
Sangamo is also progressing ST‑506, an investigational epigenetic regulator for the treatment of prion disease. Company communications explain that ST‑506 leverages STAC‑BBB, Sangamo’s proprietary neurotropic adeno‑associated virus (AAV) capsid, to deliver therapy to the brain. Preclinical data presented by Sangamo have shown survival extension in disease mouse models and widespread brain delivery with prion reduction in non‑human primates. The company is conducting Clinical Trial Application (CTA)‑enabling activities and has discussed alignment with the U.K. Medicines and Healthcare products Regulatory Agency on chemistry, manufacturing and controls and clinical study design, with plans for a future CTA submission.
Technology platforms and collaborations
Sangamo emphasizes several technology platforms that support its pipeline. In addition to zinc finger epigenetic regulators, the company describes a capsid discovery platform that includes STAC‑BBB and the SIFTER platform, intended to expand delivery of genomic medicines to the central nervous system. Sangamo is also advancing a modular integrase (MINT) genome editing platform as part of its next‑generation genome editing efforts.
The company’s disclosures highlight that its pipeline includes multiple partnered programs and that it has entered into collaboration and license agreements involving its technologies. For example, Sangamo has announced a capsid license agreement with Eli Lilly and Company granting Lilly a worldwide exclusive license to the STAC‑BBB capsid for up to five potential central nervous system disease targets, with an upfront license fee and potential additional target fees, milestone payments and royalties described in its public communications. Sangamo has also reported revenue from collaboration and license agreements with other counterparties in its financial statements.
Regulatory status and listing
Sangamo’s common stock, with a par value of $0.01 per share, is listed on the Nasdaq Capital Market under the trading symbol SGMO, as stated in its current reports on Form 8‑K. In an 8‑K dated October 29, 2025, the company reported receiving an extension from Nasdaq to regain compliance with the minimum $1.00 per share bid price requirement for continued listing, with a 180‑day period to cure the deficiency. The company noted that the extension had no immediate effect on the listing of its common stock and that it intended to cure the bid price requirement within the specified timeframe, while acknowledging that there can be no assurance of regaining or maintaining compliance.
Business focus and financial reporting
Across its quarterly financial updates, Sangamo describes itself as operating a neurology‑focused genomic medicine business, with resources directed toward advancing isaralgagene civaparvovec toward a potential BLA submission and progressing its neurology pipeline, while seeking business development opportunities such as a potential Fabry commercialization agreement and additional collaborations for its capsid and epigenetic regulation technologies. The company regularly reports its operating expenses, research and development activities, general and administrative costs, and cash and cash equivalents in its earnings releases and related Form 8‑K filings.
Sangamo’s public communications consistently underscore that its product candidates and technologies are investigational and that forward‑looking statements about regulatory pathways, potential approvals, commercial launches, collaborations and financial runway are subject to risks and uncertainties described in its filings with the U.S. Securities and Exchange Commission.