Drug Information
| Drug General Information | Top | |||
|---|---|---|---|---|
| Drug ID |
D0TBF3
|
|||
| Drug Name |
AT-GTX-501
|
|||
| Drug Type |
Gene therapy
|
|||
| Indication | CLN6 disease [ICD-11: 5C56.1] | Phase 1/2 | [1] | |
| Company |
Amicus Therapeutics
|
|||
| Target and Pathway | Top | |||
|---|---|---|---|---|
| Target(s) | Ceroid-lipofuscinosis neuronal protein 6 (CLN6) | Target Info | . | [1] |
| References | Top | |||
|---|---|---|---|---|
| REF 1 | ClinicalTrials.gov (NCT02725580) Phase I/IIa Gene Transfer Clinical Trial for Variant Late Infantile Neuronal Ceroid Lipofuscinosis, Delivering the CLN6 Gene by Self-Complementary AAV9. U.S.National Institutes of Health. | |||
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