Drug Information
| Drug General Information | Top | |||
|---|---|---|---|---|
| Drug ID |
D3O5XK
|
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| Drug Name |
SPK-3006
|
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| Synonyms |
RG6359
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|
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| Drug Type |
Gene therapy
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| Indication | Pompe disease [ICD-11: 5C51.3; ICD-10: E74.0] | Phase 1/2 | [1] | |
| Company |
Spark Therapeutics
|
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| Target and Pathway | Top | |||
|---|---|---|---|---|
| Target(s) | Lysosomal alpha-glucosidase (GAA) | Target Info | . | [2] |
| KEGG Pathway | Galactose metabolism | |||
| Starch and sucrose metabolism | ||||
| Metabolic pathways | ||||
| Lysosome | ||||
| Pathwhiz Pathway | Galactose Metabolism | |||
| Pathway Interaction Database | Notch-mediated HES/HEY network | |||
| References | Top | |||
|---|---|---|---|---|
| REF 1 | ClinicalTrials.gov (NCT04093349) Phase 1/2, Dose-escalation Study to Evaluate the Safety, Tolerability and Efficacy of a Single Intravenous Infusion of SPK-3006 in Adults With Late-onset Pompe Disease. U.S.National Institutes of Health. | |||
| REF 2 | Clinical pipeline report, company report or official report of Spark Therapeutics | |||
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