Last update 08 May 2025

AAV9-MMAB (NIH/NCATS)

Overview

Basic Info

Drug Type
AAV based gene therapy
Synonyms
Adeno-associated virus 9 vector expressing a functional human codon optimized cDNA encoding MMAB (NIH/NCATS)
Target
MMAB
Action
modulators
Mechanism
MMAB modulators(metabolism of cobalamin associated B modulators)
Therapeutic Areas
Congenital DisordersEndocrinology and Metabolic DiseaseOther Diseases
Active Indication
Methylmalonic 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-MMAB (NIH/NCATS)
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100 Translational Medicine associated with AAV9-MMAB (NIH/NCATS)
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100 Patents (Medical) associated with AAV9-MMAB (NIH/NCATS)
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100 Deals associated with AAV9-MMAB (NIH/NCATS)
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R&D Status

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IndicationHighest PhaseCountry/LocationOrganizationDate
Methylmalonic AcidemiaPreclinical
United States
National Center for Advancing Translational SciencesNational Center for Advancing Translational Sciences
16 Oct 2024
Methylmalonic AcidemiaPreclinical
United States
National Institutes of HealthNational Institutes of Health
16 Oct 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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