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Last update 23 Jan 2025
CLN1 disease(Spark)
Last update 23 Jan 2025
Overview
Basic Info
Drug Type AAV based gene therapy |
Synonyms AAV-mediated PPT1 replacement (Spark Therapeutics) |
Target |
Mechanism PPT1 replacements(Palmitoyl-protein thioesterase 1 replacements) |
Therapeutic Areas |
Active Indication |
Inactive Indication- |
Originator Organization |
Active Organization |
Inactive Organization- |
Drug Highest PhasePreclinical |
First Approval Date- |
Regulation- |
R&D Status
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| Indication | Highest Phase | Country/Location | Organization | Date |
|---|---|---|---|---|
| Ceroid Lipofuscinosis, Neuronal, 1 | Preclinical | US | 23 Apr 2024 |
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Clinical Result
Clinical Result
Indication
Phase
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