Last update 31 Jul 2025

AAV9-hPCCB (NIH/NCATS)

Overview

Basic Info

Drug Type
AAV based gene therapy
Synonyms
Adeno-Associated Virus 9 vector expressing a wild-type cDNA encoding human Propionyl-CoA Carboxylase, subunit beta (NIH/NCATS)
Target
PCCB
Action
modulators
Mechanism
PCCB modulators(propionyl-CoA carboxylase subunit beta modulators)
Therapeutic Areas
Congenital DisordersEndocrinology and Metabolic DiseaseOther Diseases
Active Indication
Propionic Acidemia
Inactive Indication-
Originator Organization
National Center for Advancing Translational SciencesNational Center for Advancing Translational SciencesNational Institutes of HealthNational Institutes of Health
Active Organization
National 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 AAV9-hPCCB (NIH/NCATS)
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100 Translational Medicine associated with AAV9-hPCCB (NIH/NCATS)
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100 Patents (Medical) associated with AAV9-hPCCB (NIH/NCATS)
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100 Deals associated with AAV9-hPCCB (NIH/NCATS)
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R&D Status

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IndicationHighest PhaseCountry/LocationOrganizationDate
Propionic AcidemiaPreclinical
United States
National Institutes of HealthNational Institutes of Health
10 Sep 2024
Propionic AcidemiaPreclinical
United States
National Center for Advancing Translational SciencesNational Center for Advancing Translational Sciences
10 Sep 2024
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Clinical Result

Indication
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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