Last update 16 Mar 2026

AAV9-hMMACHC (NHGRI/NIH)

Overview

Basic Info

Drug Type
AAV based gene therapy
Synonyms
Adeno-Associated Virus 9 vector expressing human Metabolism of Cobalamin Associated C gene (NHGRI/NIH)
Target
MMACHC
Action
agonists
Mechanism
MMACHC agonists(metabolism of cobalamin associated C agonists)
Therapeutic Areas
Nervous System DiseasesCongenital DisordersDigestive System Disorders+ [3]
Active Indication
Methylmalonic Acidemia With HomocystinuriaMethylmalonic Acidemia
Inactive Indication-
Originator Organization
National Institutes of HealthNational Institutes of HealthNational Human Genome Research InstituteNational Human Genome Research Institute
Active Organization
National Human Genome Research InstituteNational Human Genome Research InstituteNational Institutes of HealthNational Institutes of Health
Inactive Organization-
License Organization-
Drug Highest PhasePreclinical
First Approval Date-
RegulationOrphan Drug (United States)

Related

100 Clinical Results associated with AAV9-hMMACHC (NHGRI/NIH)
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100 Translational Medicine associated with AAV9-hMMACHC (NHGRI/NIH)
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100 Patents (Medical) associated with AAV9-hMMACHC (NHGRI/NIH)
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100 Deals associated with AAV9-hMMACHC (NHGRI/NIH)
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R&D Status

10 top R&D records.
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IndicationHighest PhaseCountry/LocationOrganizationDate
Methylmalonic Acidemia With HomocystinuriaPreclinical
United States
National Human Genome Research InstituteNational Human Genome Research Institute
13 May 2025
Methylmalonic AcidemiaPreclinical
United States
National Institutes of HealthNational Institutes of Health
09 Oct 2024
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Clinical Result

Indication
Phase
Evaluation
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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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