Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
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 being developed for the treatment of Fabry disease, a rare inherited disorder. It is designed to deliver a functional copy of the GLA gene to provide a sustained source of the enzyme alpha-galactosidase A. ST-920 is not yet approved for any indication and remains in clinical development.
ST-920 targets alpha-galactosidase A (α-Gal A), the enzyme that is deficient in people with Fabry disease. By delivering the GLA gene, ST-920 aims to enable cells to produce the missing enzyme. Alpha-galactosidase A is an enzyme target, and restoring its activity is the intended mechanism of the therapy.
ST-920 is being developed by Sangamo Therapeutics, Inc., which trades on the Nasdaq under the ticker SGMO. Sangamo is a clinical-stage biotechnology company focused on genomic medicines, and ST-920 is its gene therapy candidate for Fabry disease.
ST-920 is in Phase 1 clinical development. The Phase 1 trial, known as STAAR, has been completed. ST-920 is investigational and has not been approved by the FDA or any other regulatory agency for the treatment of Fabry disease.
ST-920 has been evaluated in a single Phase 1 trial, NCT04046224, titled a Dose-Ranging Study of ST-920, an AAV2/6 Human Alpha Galactosidase A Gene Therapy in Subjects With Fabry Disease (STAAR). The trial enrolled 36 participants across the United States, Australia, Canada, Germany, Italy, Taiwan, and the United Kingdom, and it is now completed.