Last update 07 Aug 2025

AAV2/5-NPHP5 (NIH/NCATS)

Overview

Basic Info

Drug Type
AAV based gene therapy
Synonyms
Adeno-associated virus pseudotype 2/5 human Nephrocystin-5 (NIH/NCATS), NPHP5 gene therapy(National Eye Institute)
Target
IQCB1
Action
modulators
Mechanism
IQCB1 modulators(IQ motif containing B1 modulators)
Therapeutic Areas
Congenital DisordersEye DiseasesOther Diseases
Active Indication
Retinal DystrophiesLeber Congenital Amaurosis
Inactive Indication-
Originator Organization
National Center for Advancing Translational SciencesNational Center for Advancing Translational SciencesNational Institutes of HealthNational Institutes of Health
Active Organization
National Eye InstituteNational Eye InstituteNational Institutes of HealthNational Institutes of HealthNational Center for Advancing Translational SciencesNational Center for Advancing Translational Sciences
Inactive Organization-
License Organization-
Drug Highest PhasePreclinical
First Approval Date-
RegulationOrphan Drug (United States)

Related

100 Clinical Results associated with AAV2/5-NPHP5 (NIH/NCATS)
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100 Translational Medicine associated with AAV2/5-NPHP5 (NIH/NCATS)
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100 Patents (Medical) associated with AAV2/5-NPHP5 (NIH/NCATS)
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100 Deals associated with AAV2/5-NPHP5 (NIH/NCATS)
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R&D Status

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IndicationHighest PhaseCountry/LocationOrganizationDate
Retinal DystrophiesPreclinical
United States
National Institutes of HealthNational Institutes of Health
11 Sep 2024
Retinal DystrophiesPreclinical
United States
National Center for Advancing Translational SciencesNational Center for Advancing Translational Sciences
11 Sep 2024
Leber Congenital AmaurosisPreclinical
United States
National Eye InstituteNational Eye Institute
08 Sep 2022
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Clinical Result

Indication
Phase
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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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