Drug Information
Drug General Information | Top | |||
---|---|---|---|---|
Drug ID |
DY21QW
|
|||
Drug Name |
RGX-121
|
|||
Drug Type |
Gene therapy
|
|||
Indication | Mucopolysaccharidosis [ICD-11: 5C56.3; ICD-9: 277.5] | Phase 1/2 | [1] | |
Company |
REGENXBIO
|
Target and Pathway | Top | |||
---|---|---|---|---|
Target(s) | Iduronate 2-sulfatase (IDS) | Target Info | Replacement | [2] |
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 (NCT04571970) RGX-121 Gene Therapy in Children 5 Years of Age and Over With MPS II (Hunter Syndrome). U.S. National Institutes of Health. | |||
REF 2 | Clinical pipeline report, company report or official report of REGENXBIO. |
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