AAV9-based NGLY1 gene therapy (Grace Science)

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Report date: 2026/05/05 20:00:16

Base Information

Drug NameAAV9-based NGLY1 gene therapy (Grace Science)
Drug Alias NameGS-100
ClassificationCell and gene therapy > Gene therapy > Viral vector-based gene therapy > Adeno-associated virus vector > AAV9 vector
Mechanisms of ActionPeptide-N(4)-(N-acetyl-beta-glucosaminyl)asparagine amidase gene transference
TargetsPeptide-N(4)-(N-acetyl-beta-glucosaminyl)asparagine amidase
Therapy Area/IndicationsNGLY1 deficiencyGenetic Diseases, Inborn
Global Approved IndicationsN/A
Approved Indications in ChinaN/A
Original Approved Country/TerritoryN/A
Original Approved DateN/A
Country/TerritoryUnited States
CombinationsNo
Global Highest StatusPhase 3 Clinical
Highest Status in ChinaN/A
Originator CompaniesGrace Science FoundationGrace Science LLC
China Registration ClassN/A

Develop Information

Active CompaniesGrace Science FoundationGrace Science LLC
Inactive CompaniesN/A
Active IndicationsNGLY1 deficiencyGenetic Diseases, Inborn
Inactive IndicationsN/A

Other Information

ATC CodeN/A
Related LinksN/A