Last update 09 Jun 2025

RP-GRIP1

Overview

Basic Info

Drug Type
AAV based gene therapy
Synonyms
AAV.Anc80.hRPGRIP1, OT-004, RP-GRIP1
Target-
Action-
Mechanism
Gene transference(Gene transference)
Therapeutic Areas
Congenital DisordersEye DiseasesOther Diseases
Active Indication
Retinal DystrophiesLeber Congenital Amaurosis
Inactive Indication-
Originator Organization
Harvard Medical SchoolHarvard Medical School
Active Organization
Odylia TherapeuticsOdylia TherapeuticsPTC Therapeutics, Inc.PTC Therapeutics, Inc.
Inactive Organization-
License Organization
Odylia TherapeuticsOdylia TherapeuticsAndelyn Biosciences, Inc.Andelyn Biosciences, Inc.
PTC Therapeutics, Inc.PTC Therapeutics, Inc.
Drug Highest PhasePreclinical
First Approval Date-
RegulationOrphan Drug (United States)

Related

100 Clinical Results associated with RP-GRIP1
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100 Translational Medicine associated with RP-GRIP1
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100 Patents (Medical) associated with RP-GRIP1
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100 Deals associated with RP-GRIP1
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R&D Status

10 top R&D records.
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IndicationHighest PhaseCountry/LocationOrganizationDate
Retinal DystrophiesPreclinical
United States
Odylia TherapeuticsOdylia Therapeutics
01 Jun 2025
Leber Congenital AmaurosisPreclinical
United States
PTC Therapeutics, Inc.PTC Therapeutics, Inc.
01 Jul 2019
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Clinical Result

Indication
Phase
Evaluation
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Study
Phase
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Deal

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Clinical Trial

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Approval

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Regulation

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