Last update 26 Jun 2026

OPGx-003

Overview

Basic Info

Drug Type
AAV based gene therapy
Synonyms
NMNAT1 gene therapy (Opus Genetics), OPGx-NMNAT1, OPGX 003
+ [1]
Target
NMNAT1
Action
modulators
Mechanism
NMNAT1 modulators(nicotinamide nucleotide adenylyltransferase 1 modulators), Gene transference(Gene transference)
Therapeutic Areas
Congenital DisordersEye DiseasesOther Diseases
Active Indication
Leber Congenital Amaurosis
Inactive Indication-
Originator Organization
The Schepens Eye Research Institute, Inc.The Schepens Eye Research Institute, Inc.
Active Organization
Opus Genetics, Inc.Opus Genetics, Inc.
Inactive Organization-
License Organization
Harvard UniversityHarvard UniversityNational Resilience, Inc.National Resilience, Inc.Massachusetts Eye & Ear Infirmary, Inc.Massachusetts Eye & Ear Infirmary, Inc.
+ [1]
Drug Highest PhasePreclinical
First Approval Date-
Regulation-

Related

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

10 top R&D records.
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IndicationHighest PhaseCountry/LocationOrganizationDate
Leber Congenital AmaurosisPreclinical
United States
Opus Genetics, Inc.Opus Genetics, Inc.
01 Nov 2024
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Clinical Result

Indication
Phase
Evaluation
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Study
Phase
PopulationAnalyzed EnrollmentGroupResultsEvaluationPublication Date

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Deal

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Core Patent

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Clinical Trial

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Approval

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Regulation

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