Drug Information
| Drug General Information | Top | |||
|---|---|---|---|---|
| Drug ID |
DQ4TO3
|
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| Drug Name |
OTL-200
|
|||
| Synonyms |
Libmeldy
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|
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| Drug Type |
Gene therapy
|
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| Indication | Metachromatic leukodystrophy [ICD-11: 5C56.02] | Approved | [1] | |
| Company |
Orchard Therapeutics
|
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| Target and Pathway | Top | |||
|---|---|---|---|---|
| Target(s) | Cerebroside-sulfatase (ARSA) | Target Info | Replacement | [1] |
| KEGG Pathway | Sphingolipid metabolism | |||
| Lysosome | ||||
| Pathwhiz Pathway | Sphingolipid Metabolism | |||
| Reactome | Glycosphingolipid metabolism | |||
| WikiPathways | Sphingolipid metabolism | |||
| References | Top | |||
|---|---|---|---|---|
| REF 1 | Clinical pipeline report, company report or official report of Orchard Therapeutics. | |||
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