Last update 21 Nov 2024
AAV9-hCLN7 (Elpida Therapeutics)
Last update 21 Nov 2024
Overview
Basic Info
Drug Type AAV based gene therapy |
Synonyms Adeno-associated viral vector serotype 9 encoding a codon-optimized human ceroid neuronal lipofuscinosis type 7 (CLN7) transgene |
Target |
Mechanism MFSD8 modulators(Major facilitator superfamily domain-containing protein 8 modulators) |
Therapeutic Areas |
Active Indication |
Inactive Indication- |
Originator Organization |
Active Organization |
Inactive Organization- |
Drug Highest PhasePreclinical |
First Approval Date- |
RegulationOrphan Drug (US) |
R&D Status
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| Indication | Highest Phase | Country/Location | Organization | Date |
|---|---|---|---|---|
| Neuronal Ceroid-Lipofuscinoses | Preclinical | US | 29 Oct 2024 |
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