Last update 27 Dec 2024
VGR-R01
Last update 27 Dec 2024
Overview
Basic Info
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- |
RegulationOrphan Drug (US), Breakthrough Therapy (CN) |
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Related
6
Clinical Trials associated with VGR-R01A Multi-Center, Randomized, Controlled Phase III Clinical Study to Evaluate the Efficacy and Safety of VGR-R01 in Subjects with Bietti Crystalline Dystrophy
VGR-R01 治疗结晶样视网膜变性（BCD）的 I 期临床研究
[Translation] Phase I clinical study of VGR-R01 for the treatment of BCD
A Phase 1 Study to Assess the Safety and Tolerability of VGR-R01 in Patients With Bietti Crystalline Dystrophy
100 Clinical Results associated with VGR-R01
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100 Translational Medicine associated with VGR-R01
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100 Patents (Medical) associated with VGR-R01
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100 Deals associated with VGR-R01
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R&D Status
10 top R&D records. to view more data
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| Indication | Highest Phase | Country/Location | Organization | Date |
|---|---|---|---|---|
| Bietti Crystalline Dystrophy | Phase 3 | CN | 25 Nov 2024 |
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Clinical Result
Clinical Result
Indication
Phase
Evaluation
View All Results
NEWS Manual  | Not Applicable | 12 | VGR-R01低剂量 | vmvvqetnkq(wpbzeoajov) = hisdhevwye hiqwagyphr (gpplwlncdj ) | Positive | 10 May 2024 | |
VGR-R01中剂量 | vmvvqetnkq(wpbzeoajov) = npruidmgjf hiqwagyphr (gpplwlncdj ) | ||||||
NCT05694598 (ASGCT2024) Manual | Phase 1/2 | Bietti Crystalline Dystrophy CYP4V2 biallelic mutation | 12 | VGR-R01 | qxtpntmaqc(ovzeqhruwg) = All patients experienced a treatment-emergent adverse event (TEAE) but just one eye inflammation was related to VGR-R01. rujidlfijy (uzrxgafcrq ) | Positive | 07 May 2024 |
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