Drug Information
| Drug General Information | Top | |||
|---|---|---|---|---|
| Drug ID |
DX36OY
|
|||
| Drug Name |
RP-A501
|
|||
| Drug Type |
Gene therapy
|
|||
| Indication | Type 2 glycogen storage disease [ICD-11: 5C51.3; ICD-10: E74.0; ICD-9: 271] | Phase 1 | [1] | |
| Company |
Rocket Pharmaceuticals
|
|||
| Target and Pathway | Top | |||
|---|---|---|---|---|
| Target(s) | Lysosome-associated membrane glycoprotein 2 (LAMP2) | Target Info | Replacement | [1] |
| References | Top | |||
|---|---|---|---|---|
| REF 1 | ClinicalTrials.gov (NCT03882437) Gene Therapy for Male Patients With Danon Disease (DD) Using RP-A501; AAV9.LAMP2B. U.S. National Institutes of Health. | |||
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