Last update 17 May 2025

AAV9 vector-IDUA gene (NeuroGT)

Overview

Basic Info

Drug Type
AAV based gene therapy
Synonyms
scAAV-∆h IDUA op, Self-complementary adeno-associated virus serotype 9 vector harboring a miniaturized codon-optimized human IDUA gene
Target
IDUA
Action
stimulants
Mechanism
IDUA gene stimulants(Alpha-L-iduronidase gene stimulants)
Therapeutic Areas
Congenital DisordersEndocrinology and Metabolic DiseaseSkin and Musculoskeletal Diseases+ [1]
Active Indication
Mucopolysaccharidosis I
Inactive Indication-
Originator Organization
NeuroGT Inc.
Active Organization
The University of North Carolina at Chapel HillThe University of North Carolina at Chapel HillNeuroGT Inc.
Inactive Organization-
License Organization-
Drug Highest PhasePreclinical
First Approval Date-
RegulationOrphan Drug (United States)

Related

100 Clinical Results associated with AAV9 vector-IDUA gene (NeuroGT)
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100 Translational Medicine associated with AAV9 vector-IDUA gene (NeuroGT)
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100 Patents (Medical) associated with AAV9 vector-IDUA gene (NeuroGT)
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100 Deals associated with AAV9 vector-IDUA gene (NeuroGT)
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R&D Status

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IndicationHighest PhaseCountry/LocationOrganizationDate
Mucopolysaccharidosis IPreclinical
United States
The University of North Carolina at Chapel HillThe University of North Carolina at Chapel Hill
07 May 2024
Mucopolysaccharidosis IPreclinical
United States
NeuroGT Inc.
07 May 2024
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Clinical Result

Indication
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Approval

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Regulation

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