Last update 08 May 2025

AAV-GAMT

Overview

Basic Info

Drug Type
AAV based gene therapy
Synonyms
Adeno-associated vector expressing human codon-optimized guanidinoacetate N-methyltransferase under a ubiquitous-expressing promoter
Target
GAMT
Action
agonists
Mechanism
GAMT agonists(guanidinoacetate N-methyltransferase agonists)
Therapeutic Areas
Nervous System DiseasesOther Diseases
Active Indication
Guanidinoacetate Methyltransferase Deficiency
Inactive Indication-
Originator Organization
UCLA Medical CenterUCLA Medical Center
Active Organization
UCLA Medical CenterUCLA Medical Center
Inactive Organization-
License Organization-
Drug Highest PhasePreclinical
First Approval Date-
RegulationOrphan Drug (United States)

Related

100 Clinical Results associated with AAV-GAMT
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100 Translational Medicine associated with AAV-GAMT
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100 Patents (Medical) associated with AAV-GAMT
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100 Deals associated with AAV-GAMT
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R&D Status

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IndicationHighest PhaseCountry/LocationOrganizationDate
Guanidinoacetate Methyltransferase DeficiencyPreclinical
United States
UCLA Medical CenterUCLA Medical Center
18 Feb 2025
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Clinical Result

Indication
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Approval

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Regulation

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