Last update 08 May 2025

AAV8-GALNS (NCATS/NIH)

Overview

Basic Info

Drug Type
AAV based gene therapy
Synonyms
adeno-associated virus 8 vector expressing functional human cDNA encoding the N-acetylgalactosamine-6-sulfate sulfatase
Target
GALNS
Action-
Mechanism
GALNS replacements(galactosamine (N-acetyl)-6-sulfatase replacements)
Therapeutic Areas
Congenital DisordersEndocrinology and Metabolic DiseaseSkin and Musculoskeletal Diseases+ [1]
Active Indication
Mucopolysaccharidosis IV
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 AAV8-GALNS (NCATS/NIH)
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100 Translational Medicine associated with AAV8-GALNS (NCATS/NIH)
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100 Patents (Medical) associated with AAV8-GALNS (NCATS/NIH)
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100 Deals associated with AAV8-GALNS (NCATS/NIH)
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R&D Status

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IndicationHighest PhaseCountry/LocationOrganizationDate
Mucopolysaccharidosis IVPreclinical
United States
National Institutes of HealthNational Institutes of Health
20 Sep 2024
Mucopolysaccharidosis IVPreclinical
United States
National Center for Advancing Translational SciencesNational Center for Advancing Translational Sciences
20 Sep 2024
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Clinical Result

Indication
Phase
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Clinical Trial

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Approval

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Regulation

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