AAV-GAA gene therapy (Duke University/Genethon/Actus Therapeutics)

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Report date: 2023/10/26 16:41:08

Base Information

Drug NameAAV-GAA gene therapy (Duke University/Genethon/Actus Therapeutics)
Drug Alias NameAdeno-associated virus based gene therapy (Duke University/Genethon/Actus Therapeutics)AAV2/8-LsPhGAAACTUS-101AAV8-SEC-GAA
ClassificationCell and gene therapy > Gene therapy > Viral vector-based gene therapy > Adeno-associated virus vector > AAV2/8 vector
Mechanisms of ActionGAA gene transference
TargetsAcidic alpha-glucosidase
Therapy Area/IndicationsGlycogen Storage Disease Type II
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 2 Clinical
Highest Status in ChinaN/A
Originator CompaniesActus TherapeuticsDuke University Medical CenterNational Institutes Of Health
China Registration ClassN/A

Develop Information

Active CompaniesAskBio IncNational Institutes Of HealthDuke University Medical Center +[4] 
Inactive CompaniesN/A
Active IndicationsGlycogen Storage Disease Type II
Inactive IndicationsN/A

Other Information

ATC CodeN/A
Related LinksN/A