Approval Probability
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ST-920 · 1 trial · 1 indication
All incidences of Treatment-Emergent Adverse Events (TEAEs) in subjects who receive ST-920 as assessed by Common Terminology Criteria for Adverse Events (CTCAE)
Incidences of Treatment-Emergent Adverse Events (TEAEs) directly related to ST-920 in subjects who receive ST-920 as assessed by Common Terminology Criteria for Adverse Events (CTCAE)
All incidences of serious Treatment-Emergent Adverse Events (TEAEs) in subjects who receive ST-920 as assessed by Common Terminology Criteria for Adverse Events (CTCAE)
All incidences of Treatment-Emergent Adverse Events (TEAEs) that lead to study discontinuation or withdrawal in subjects who receive ST-920 as assessed by Common Terminology Criteria for Adverse Events (CTCAE)
| Arm | Type | Description |
|---|---|---|
| Sequential dose escalation | EXPERIMENTAL | ST-920 is administered as a single infusion: 1. Cohort 1: 0.5e13 vg/kg 2. Cohort 2: 1.0e13 vg/kg 3. Cohort 3: 3.0e13 vg/kg 4. Cohort 4: 5.0e13 vg/kg |
| Expansion Cohorts | EXPERIMENTAL | 1. Anti Alpha-Gal A Antibody Positive Cohort 2. Anti Alpha-Gal A Antibody Negative Cohort 3. Female Cohort 4. Renal Cohort 5. Cardiac Cohort |
| Name | Type | Description |
|---|---|---|
| ST-920 | BIOLOGICAL | Single dose of investigational product ST-920 |
Inclusion Criteria: * ≥ 18 years of age * Documented diagnosis of Fabry disease * One or more of the following symptoms: i) cornea verticillata, ii) acroparesthesia, iii) anhidrosis, iv) angiokeratoma * Subject must be fully vaccinated (as per the Centers for Disease Control and Prevention (CDC) de...
ST-920 is an investigational gene therapy for Fabry disease developed by Sangamo Therapeutics. It is designed to deliver a functional copy of the gene for alpha-galactosidase A (alpha-Gal A), the enzyme that is deficient in people with Fabry disease. ST-920 is being studied as a one-time treatment approach for this rare inherited condition.
ST-920 targets alpha-galactosidase A (alpha-Gal A), the enzyme that is missing or deficient in Fabry disease. The therapy uses an AAV2/6 vector to deliver the human alpha-galactosidase A gene, with the goal of enabling patients' cells to produce the enzyme themselves. Alpha-Gal A is an enzyme target rather than a receptor or kinase.
ST-920 is being developed by Sangamo Therapeutics, Inc., a clinical-stage biotechnology company. Sangamo's stock ticker is SGMOQ. The company is advancing ST-920 as a gene therapy candidate for Fabry disease, a rare lysosomal storage disorder.
ST-920 is in Phase 1/2 clinical development for Fabry disease. It is an investigational therapy and has not been approved by the FDA. Its clinical program includes a completed Phase 1/2 dose-ranging study, the STAAR trial, which enrolled 36 participants across the United States, Australia, Canada, Germany, Italy, Taiwan, and the United Kingdom.
ST-920 has been studied in the STAAR trial, registered as NCT04046224, a Phase 1/2 dose-ranging study of the AAV2/6 human alpha-galactosidase A gene therapy in adults with Fabry disease. The trial enrolled 36 participants, was controlled in design, and has completed. It included sites in the United States, Australia, Canada, Germany, Italy, Taiwan, and the United Kingdom.
ST-920 is being developed as a gene therapy treatment for Fabry disease, a rare inherited disorder caused by deficiency of the enzyme alpha-galactosidase A. The goal of the therapy is to provide patients with a working copy of the alpha-galactosidase A gene so their cells can produce the enzyme. It remains investigational and is not approved for use.