Last update 27 Apr 2026

RRG-003

Overview

Basic Info

Drug Type
AAV based gene therapy
Synonyms
AAV1-hOTOF, Dual recombinant adeno-associated viral vectors encoding the human otoferlin gene, RRG-OTOF
+ [2]
Target
OTOF
Action
modulators
Mechanism
OTOF modulators(otoferlin modulators)
Therapeutic Areas
Nervous System DiseasesOtorhinolaryngologic DiseasesOther Diseases
Active Indication
DeafnessDeafness, Autosomal Recessive 9Hearing Loss
Inactive Indication-
Originator Organization
Shanghai Refreshgene Technology Co., Ltd.Shanghai Refreshgene Technology Co., Ltd.
Active Organization
Shanghai Refreshgene Technology Co., Ltd.Shanghai Refreshgene Technology Co., Ltd.Fudan University Affiliated Eye & Ent Hospital
Inactive Organization-
License Organization
Harvard UniversityHarvard UniversityShanghai Refreshgene Technology Co., Ltd.Shanghai Refreshgene Technology Co., Ltd.Fudan University Affiliated Eye & Ent Hospital
Drug Highest PhasePhase 1
First Approval Date-
RegulationOrphan Drug (United States)
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Related

100 Clinical Results associated with RRG-003
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100 Translational Medicine associated with RRG-003
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100 Patents (Medical) associated with RRG-003
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100 Deals associated with RRG-003
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R&D Status

10 top R&D records.
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IndicationHighest PhaseCountry/LocationOrganizationDate
DeafnessPhase 1
China
Shanghai Refreshgene Technology Co., Ltd.Shanghai Refreshgene Technology Co., Ltd.
17 Nov 2022
DeafnessPhase 1
China
Fudan University Affiliated Eye & Ent Hospital
17 Nov 2022
Deafness, Autosomal Recessive 9Phase 1
China
Fudan University Affiliated Eye & Ent Hospital
01 Sep 2022
Hearing LossIND Approval
United States
Shanghai Refreshgene Technology Co., Ltd.Shanghai Refreshgene Technology Co., Ltd.
-
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Clinical Result

Indication
Phase
Evaluation
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Study
Phase
PopulationAnalyzed EnrollmentGroupResultsEvaluationPublication Date
NEWS
ManualManual
Not Applicable
Deafness
5
oniihseuga(ydfsmbhwnf) = ymcpjgfbjq sjrasnobyk (wpbhwlgfky )
Positive
31 Oct 2023
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Clinical Trial

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Approval

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Regulation

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