Last update 29 Nov 2024

VGR-R01

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)
Inactive Indication-
Originator Organization
Active Organization
Inactive Organization-
Drug Highest PhasePhase 3
First Approval Date-
RegulationBreakthrough Therapy (CN), Orphan Drug (US)
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R&D Status

10 top R&D records.
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IndicationHighest PhaseCountry/LocationOrganizationDate
Bietti Crystalline DystrophyPhase 3
CN
25 Nov 2024
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Clinical Result

Indication
Phase
Evaluation
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Study
Phase
PopulationAnalyzed EnrollmentGroupResultsEvaluationPublication Date
NEWS
ManualManual
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 )
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
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