Drug Information
| Drug General Information | Top | |||
|---|---|---|---|---|
| Drug ID |
DF7R6C
|
|||
| Drug Name |
AT-GTX-502
|
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| Drug Type |
Gene therapy
|
|||
| Indication | CLN3 Batten disease [ICD-11: 5C56] | Phase 1/2 | [1] | |
| Company |
Amicus Therapeutics Philadelphia, PA
|
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| Target and Pathway | Top | |||
|---|---|---|---|---|
| Target(s) | Battenin (CLN3) | Target Info | . | [1] |
| References | Top | |||
|---|---|---|---|---|
| REF 1 | ClinicalTrials.gov (NCT03770572) Phase I/IIa Gene Transfer Clinical Trial for Juvenile Neuronal Ceroid Lipofuscinosis, Delivering the CLN3 Gene by Self-Complementary AAV9. U.S.National Institutes of Health. | |||
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