Drug Information
| Drug General Information | Top | |||
|---|---|---|---|---|
| Drug ID |
DY9DX8
|
|||
| Drug Name |
SB-913
|
|||
| Drug Type |
Gene therapy
|
|||
| Indication | Mucopolysaccharidosis [ICD-11: 5C56.3; ICD-9: 277.5] | Phase 1/2 | [1] | |
| Company |
Sangamo Therapeutics
|
|||
| Target and Pathway | Top | |||
|---|---|---|---|---|
| Target(s) | Iduronate 2-sulfatase (IDS) | Target Info | Replacement | [1] |
| BioCyc | Dermatan sulfate degradation (metazoa) | |||
| KEGG Pathway | Glycosaminoglycan degradation | |||
| Metabolic pathways | ||||
| Lysosome | ||||
| Reactome | HS-GAG degradation | |||
| CS/DS degradation | ||||
| WikiPathways | Glycosaminoglycan metabolism | |||
| References | Top | |||
|---|---|---|---|---|
| REF 1 | ClinicalTrials.gov (NCT03041324) Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-913 in Subjects With MPS II. U.S. National Institutes of Health. | |||
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