Drug Information
| Drug General Information | Top | |||
|---|---|---|---|---|
| Drug ID |
DE3M8U
|
|||
| Drug Name |
ST-920
|
|||
| Drug Type |
Gene therapy
|
|||
| Indication | Fabry disease [ICD-11: 5C56.01; ICD-9: 272.7] | Phase 1/2 | [1] | |
| Company |
Sangamo Therapeutics
|
|||
| Target and Pathway | Top | |||
|---|---|---|---|---|
| Target(s) | Alpha-galactosidase A (GLA) | Target Info | . | [2] |
| KEGG Pathway | Galactose metabolism | |||
| Glycerolipid metabolism | ||||
| Sphingolipid metabolism | ||||
| Glycosphingolipid biosynthesis - globo series | ||||
| Lysosome | ||||
| Pathwhiz Pathway | Sphingolipid Metabolism | |||
| Galactose Metabolism | ||||
| Reactome | Glycosphingolipid metabolism | |||
| WikiPathways | Sphingolipid metabolism | |||
| References | Top | |||
|---|---|---|---|---|
| REF 1 | ClinicalTrials.gov (NCT04046224) A Phase I/II, Multicenter, Open-Label, Single-Dose, Dose-Ranging Study to Assess the Safety and Tolerability of ST-920, an AAV2/6 Human Alpha Galactosidase A Gene Therapy, in Subjects With Fabry Disease (STAAR). U.S.National Institutes of Health. | |||
| REF 2 | AAV2/6 Gene Therapy in a Murine Model of Fabry Disease Results in Supraphysiological Enzyme Activity and Effective Substrate Reduction. Mol Ther Methods Clin Dev. 2020 Jul 9;18:607-619. | |||
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