Last update 21 Nov 2024

AAV9 vector-IDUA gene (NeuroGT)

Overview

Basic Info

Drug Type
AAV based gene therapy
Synonyms
Self-complementary adeno-associated virus serotype 9 vector harboring a miniaturized codon-optimized human IDUA gene, scAAV-∆h IDUA op
Target
IDUA
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-
Drug Highest PhasePreclinical
First Approval Date-
RegulationOrphan Drug (US)

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
US
NeuroGT Inc.
07 May 2024
Mucopolysaccharidosis IPreclinical
US
The University of North Carolina at Chapel HillThe University of North Carolina at Chapel Hill
07 May 2024
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Clinical Result

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Approval

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Regulation

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