Drug Type AAV based gene therapy |
Synonyms Recombinant AAV9 viral vector containing the transgene encoding the human WW domain-containing oxidoreductase (WWOX) protein, under the control of a human Synapsin I promoter |
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Drug Highest PhasePreclinical |
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RegulationOrphan Drug (United States) |
Indication | Highest Phase | Country/Location | Organization | Date |
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Epileptic encephalopathy | Preclinical | United States | 07 Dec 2023 |