Drug Information
| Drug General Information | Top | |||
|---|---|---|---|---|
| Drug ID |
D09ROP
|
|||
| Drug Name |
AMT-130
|
|||
| Drug Type |
Gene therapy
|
|||
| Indication | Huntington disease [ICD-11: 8A01.10; ICD-10: G10; ICD-9: 294.1, 333.4] | Phase 1/2 | [1] | |
| Company |
uniQure Biopharma
|
|||
| Target and Pathway | Top | |||
|---|---|---|---|---|
| Target(s) | Huntingtin (HTT) | Target Info | . | [1] |
| References | Top | |||
|---|---|---|---|---|
| REF 1 | ClinicalTrials.gov (NCT05243017) A Phase Ib/II Study to Explore Safety, Tolerability, and Efficacy Signals of Multiple Ascending Doses of Striatally-Administered rAAV5-miHTT Total Huntingtin Gene (HTT) Lowering Therapy (AMT 130) in Early Manifest Huntington's Disease. U.S.National Institutes of Health. | |||
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