Last update 08 May 2025

rAAV2/4.hRPE65(Nantes University Hospital)

Overview

Basic Info

Drug Type
AAV based gene therapy
Synonyms-
Target-
Action-
Mechanism-
Therapeutic Areas
Congenital DisordersEye DiseasesOther Diseases
Active Indication-
Inactive Indication
Amaurosis Congenita of Leber, Type 2Biallelic RPE65 mutation associated retinal dystrophyLeber Congenital Amaurosis+ [1]
Originator Organization
Nantes University HospitalNantes University Hospital
Active Organization-
Inactive Organization
Nantes University HospitalNantes University HospitalBeacon Therapeutics (USA), Inc.Beacon Therapeutics (USA), Inc.
License Organization-
Drug Highest PhasePendingPhase 1/2
First Approval Date-
Regulation-
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Related

1
Clinical Trials associated with rAAV2/4.hRPE65(Nantes University Hospital)
NCT01496040
/ CompletedPhase 1/2IIT
Prospective Monocentric Open Label Non Randomized Uncontrolled Phase I/II Clinical Gene Therapy Protocol for the Treatment of Retinal Dystrophy Caused by Defects in RPE65
100 Clinical Results associated with rAAV2/4.hRPE65(Nantes University Hospital)
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100 Translational Medicine associated with rAAV2/4.hRPE65(Nantes University Hospital)
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100 Patents (Medical) associated with rAAV2/4.hRPE65(Nantes University Hospital)
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100 Deals associated with rAAV2/4.hRPE65(Nantes University Hospital)
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R&D Status

10 top R&D records.
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IndicationHighest PhaseCountry/LocationOrganizationDate
Retinal Dystrophy, Early Onset SeverePhase 2
France
Nantes University HospitalNantes University Hospital
30 Sep 2011
Biallelic RPE65 mutation associated retinal dystrophyPhase 2
France
Nantes University HospitalNantes University Hospital
01 Sep 2011
Leber Congenital AmaurosisPhase 2
France
Nantes University HospitalNantes University Hospital
01 Sep 2011
Amaurosis Congenita of Leber, Type 2Phase 2
United States
Beacon Therapeutics (USA), Inc.Beacon Therapeutics (USA), Inc.
17 Jun 2009
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Clinical Result

Indication
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Approval

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Regulation

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