Drug Type AAV based gene therapy |
Synonyms |
Target |
Mechanism GBA stimulants(Beta-glucocerebrosidase stimulants), Gene transference |
Therapeutic Areas |
Active Indication |
Inactive Indication- |
Originator Organization |
Active Organization |
Inactive Organization- |
Drug Highest PhaseEarly Phase 1 |
First Approval Date- |
RegulationRare Pediatric Disease (US) |
Indication | Highest Phase | Country/Location | Organization | Date |
---|---|---|---|---|
Gaucher Disease, Type 2 | Phase 1 | CN | 01 Mar 2023 |
Not Applicable | - | lkgjkqhvhj(eeipizzxgr) = dflnwknhbr jctpitgufk (ccczubaeqm ) View more | - | 07 May 2024 |