Drug Type AAV based gene therapy |
Synonyms LYS GM101, LYS-GM101 |
Target |
Mechanism β-galactosidase modulators(Beta-galactosidase modulators) |
Therapeutic Areas |
Active Indication- |
Inactive Indication |
Originator Organization |
Active Organization- |
Inactive Organization |
Drug Highest PhasePendingPhase 1/2 |
First Approval Date- |
RegulationFast Track (US), Orphan Drug (US), Rare Pediatric Disease (US) |
Indication | Highest Phase | Country/Location | Organization | Date |
---|---|---|---|---|
Gangliosidosis, GM1 | Phase 2 | US | 11 May 2021 | |
Gangliosidosis, GM1 | Phase 2 | FR | 11 May 2021 | |
Gangliosidosis, GM1 | Phase 2 | GB | 11 May 2021 |