Drug Information
| Drug General Information | Top | |||
|---|---|---|---|---|
| Drug ID |
DFV71O
|
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| Drug Name |
AAV2/8-LSPhGAA
|
|||
| Synonyms |
ACTUS 101
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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 | [1] | |
| Company |
Actus Therapeutics
|
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| Target and Pathway | Top | |||
|---|---|---|---|---|
| Target(s) | Alpha-glucosidase (GLA) | Target Info | . | [1] |
| References | Top | |||
|---|---|---|---|---|
| REF 1 | ClinicalTrials.gov (NCT03533673) AAV2/8-LSPhGAA in Late-Onset Pompe Disease. U.S. National Institutes of Health. | |||
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