Drug Type AAV based gene therapy |
Synonyms MECP2 duplication syndrome (Cholgene) |
Target |
Mechanism MECP2 modulators(Methyl-CpG-binding protein 2 modulators) |
Therapeutic Areas |
Active Indication |
Inactive Indication- |
Originator Organization |
Active Organization |
Inactive Organization- |
Drug Highest PhasePreclinical |
First Approval Date- |
RegulationRare Pediatric Disease (US), Orphan Drug (EU) |
Indication | Highest Phase | Country/Location | Organization | Date |
---|---|---|---|---|
Lubs X-Linked Mental Retardation Syndrome | Preclinical | CN | 06 Sep 2023 |