Drug Type AAV based gene therapy |
Synonyms A non-replicating recombinant adeno-associated virus vector serotype 8 delivering CYP4V2 gene, VGR R01, VGRR01 |
Target |
Mechanism CYP4V2 replacements(cytochrome P450 family 4 subfamily V member 2 replacements) |
Therapeutic Areas |
Active Indication |
Inactive Indication- |
Originator Organization |
Active Organization |
Inactive Organization- |
Drug Highest PhasePhase 3 |
First Approval Date- |
RegulationBreakthrough Therapy (CN), Orphan Drug (US) |
Indication | Highest Phase | Country/Location | Organization | Date |
---|---|---|---|---|
Bietti Crystalline Dystrophy | Phase 3 | CN | 25 Nov 2024 |
NEWS Manual | Not Applicable | 12 | VGR-R01低剂量 | gomtlenybh(dbznhjtjta) = iebtqkweex ihhyqiwshk (tdipcvytwn ) | Positive | 10 May 2024 | |
VGR-R01中剂量 | gomtlenybh(dbznhjtjta) = yvehhtsimc ihhyqiwshk (tdipcvytwn ) | ||||||
NCT05694598 (ASGCT2024) Manual | Phase 1/2 | Bietti Crystalline Dystrophy CYP4V2 biallelic mutation | 12 | VGR-R01 | rbezmjgtuh(mljxtsnxfu) = All patients experienced a treatment-emergent adverse event (TEAE) but just one eye inflammation was related to VGR-R01. zxkfybdzzo (qfvxyrxnbx ) | Positive | 07 May 2024 |