Drug Information
| Drug General Information | Top | |||
|---|---|---|---|---|
| Drug ID |
D5DAR1
|
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| Drug Name |
LYS-SAF302
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| Synonyms |
SRP-9002
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|
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| Drug Type |
Gene therapy
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| Indication | Mucopolysaccharidosis [ICD-11: 5C56.3; ICD-9: 277.5] | Phase 2/3 | [1] | |
| Company |
Lysogene; Sarepta Therapeutics
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| Target and Pathway | Top | |||
|---|---|---|---|---|
| Target(s) | N-sulphoglucosamine sulphohydrolase (SGSH) | Target Info | Replacement | [2] |
| KEGG Pathway | Glycosaminoglycan degradation | |||
| Metabolic pathways | ||||
| Lysosome | ||||
| Reactome | HS-GAG degradation | |||
| WikiPathways | Glycosaminoglycan metabolism | |||
| References | Top | |||
|---|---|---|---|---|
| REF 1 | ClinicalTrials.gov (NCT03612869) Study of AAVrh10-h.SGSH Gene Therapy in Patients With Mucopolysaccharidosis Type IIIA (MPS IIIA) (AAVance). U.S. National Institutes of Health. | |||
| REF 2 | Clinical pipeline report, company report or official report of Lysogene. | |||
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