This section highlights FDA-related milestones and regulatory updates for drugs developed by Sangamo Therapeutics (SGMO).
Over the past two years, Sangamo Therapeutics has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as
ST-503 and ST-920. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
ST-503 - FDA Regulatory Timeline and Events
ST-503 is a drug developed by Sangamo Therapeutics for the following indication: For the Treatment of Idiopathic Small Fiber Neuropathy, a Type of Chronic Neuropathic Pain.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- ST-503
- Announced Date:
- November 19, 2024
- Indication:
- For the Treatment of Idiopathic Small Fiber Neuropathy, a Type of Chronic Neuropathic Pain
Announcement
Sangamo Therapeutics, Inc announced that the U.S. Food and Drug Administration (FDA) has cleared the investigational new drug (IND) application for its ST-503 program, an investigational epigenetic regulator for the treatment of intractable pain due to idiopathic small fiber neuropathy (iSFN), a type of chronic neuropathic pain.
AI Summary
Sangamo Therapeutics announced that the FDA has cleared its investigational new drug (IND) application for the ST-503 program. ST-503 is an investigational epigenetic regulator designed to treat intractable pain caused by idiopathic small fiber neuropathy (iSFN), a type of chronic neuropathic pain that severely affects patients. The therapy works by targeting the SCN9A gene, reducing the expression of the Nav1.7 sodium channel, which plays a critical role in pain signaling. Preclinical studies have shown that a single intrathecal dose of ST-503 significantly reduced pain symptoms in animal models without causing off-target effects.
Sangamo plans to start enrolling patients in a Phase 1/2 clinical study in mid-2025 to assess the safety, tolerability, and preliminary efficacy of ST-503. This milestone offers hope for patients suffering from debilitating, chronic neuropathic pain with few effective treatment options.
Read Announcement
ST-920 - FDA Regulatory Timeline and Events
ST-920 is a drug developed by Sangamo Therapeutics for the following indication: FabryDisease.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- ST-920
- Announced Date:
- June 24, 2025
- Indication:
- FabryDisease
Announcement
Sangamo Therapeutics, Inc announced positive topline results from the registrational Phase 1/2 STAAR study evaluating isaralgagene civaparvovec, or ST-920, a wholly owned investigational gene therapy for the treatment of adults with Fabry disease.
AI Summary
Sangamo Therapeutics, Inc. announced positive topline results from its Phase 1/2 STAAR study evaluating isaralgagene civaparvovec (ST-920), an investigational gene therapy for adults with Fabry disease. The study showed a positive mean annualized eGFR slope of 1.965 mL/min/1.73m²/year at 52 weeks, which the FDA has agreed will serve as an intermediate clinical endpoint under Accelerated Approval. Among patients with 104 weeks of follow-up, similar improvements were observed, supporting the potential of ST-920 as a one-time, durable treatment for Fabry disease. The therapy demonstrated a favorable safety and tolerability profile with mostly mild side effects. Based on these encouraging results, Sangamo plans to submit a Biologics License Application (BLA) as early as the first quarter of 2026, aiming to offer a new option for managing the underlying pathology of Fabry disease.
Read Announcement- Drug:
- ST-920
- Announced Date:
- May 6, 2025
- Indication:
- FabryDisease
Announcement
Sangamo Therapeutics, Inc announced important derisking events in the pathway to a planned BLA submission for isaralgagene civaparvovec, or ST-920, its wholly owned gene therapy product candidate for the treatment of Fabry disease.
AI Summary
Sangamo Therapeutics, Inc. announced important progress on the path to filing a Biologics License Application (BLA) for its gene therapy candidate, isaralgagene civaparvovec (ST‑920), which is designed to treat Fabry disease. All patients in the Phase 1/2 STAAR study have reached the 52‑week follow-up milestone, a key requirement by the FDA’s Accelerated Approval pathway. Preliminary data from these 32 patients show that the positive trend in kidney function, as measured by the mean eGFR slope, continues to hold, supporting the potential efficacy of the treatment.
Additionally, a recent productive Type B Chemistry, Manufacturing, and Controls meeting with the FDA has provided a clear pathway for refining the manufacturing process and planning for process validation. These derisking milestones bolster the company’s timeline toward a planned BLA submission in early 2026, with a pivotal data readout expected by the end of the second quarter of 2025.
Read Announcement- Drug:
- ST-920
- Announced Date:
- February 6, 2025
- Indication:
- FabryDisease
Announcement
Sangamo Therapeutics, Inc announced updated data from the Phase 1/2 STAAR study evaluating isaralgagene civaparvovec, or ST-920, a wholly owned gene therapy product candidate for the treatment of Fabry disease.
AI Summary
Sangamo Therapeutics recently shared promising updated data from the Phase 1/2 STAAR study for its gene therapy candidate ST-920, which is being developed to treat Fabry disease. The study showed that patients experienced sustained benefits, with the longest treated patient maintaining elevated levels of the enzyme alpha-galactosidase A (α-Gal A) for nearly four years. In addition, 23 patients who reached at least one year of follow-up demonstrated a positive annualized improvement in kidney function as measured by eGFR. Significantly, all 18 patients who were receiving enzyme replacement therapy (ERT) were successfully withdrawn from ERT and have remained off it. These encouraging results support the potential of ST-920 as a one-time, durable treatment option that could improve patient outcomes in Fabry disease.
Read Announcement- Drug:
- ST-920
- Announced Date:
- February 6, 2025
- Estimated Event Date Range:
- July 1, 2025 - December 31, 2025
- Target Action Date:
- 2025-H2
- Indication:
- FabryDisease
Announcement
Sangamo Therapeutics, Inc announced that Biologics License Application (BLA) submission to the U.S. Food and Drug Administration (FDA) anticipated in second half of 2025
AI Summary
Sangamo Therapeutics, Inc. recently announced encouraging progress with its gene therapy candidate, ST-920, for treating Fabry disease. The company revealed that data from its ongoing Phase 1/2 STAAR study will be available in the first half of 2025, supporting key measures of kidney function and patient safety. Based on these promising results, Sangamo plans to submit a Biologics License Application (BLA) to the U.S. Food and Drug Administration in the second half of 2025. This potential submission is part of an Accelerated Approval pathway that aims to expedite the process for bringing one-time, durable treatments to patients. Along with moving forward with regulatory plans, Sangamo is also pursuing business development discussions for a possible collaboration on ST-920, reflecting growing optimism about the therapy’s benefits and its impact on Fabry disease.
Read Announcement- Drug:
- ST-920
- Announced Date:
- February 6, 2025
- Estimated Event Date Range:
- January 1, 2025 - June 30, 2025
- Target Action Date:
- 2025-H1
- Indication:
- FabryDisease
Announcement
Sangamo Therapeutics, Inc. announced Data to support Accelerated Approval pathway expected in first half of 2025
AI Summary
Sangamo Therapeutics announced that data supporting the Accelerated Approval pathway for its gene therapy candidate, ST-920, are expected in the first half of 2025. This key data, specifically the 52‐week eGFR slope from the Phase 1/2 STAAR study, will serve as the primary efficacy endpoint in its regulatory submission to the FDA. The positive outcomes highlight improvements in kidney function among patients, reinforcing ST-920's potential as a one-time, durable treatment for Fabry disease.
Looking ahead, the company anticipates submitting a Biologics License Application in the second half of 2025. Sangamo’s continued progress in its clinical development, along with ongoing partnership discussions for ST-920, underlines its commitment to advancing innovative treatments that can significantly enhance patient outcomes.
Read Announcement
