AAV-vector based GBA1 replacement therapy (Sanofi)

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Report date: 2025/06/11 11:02:35

Base Information

Drug NameAAV-vector based GBA1 replacement therapy (Sanofi)
Drug Alias NameGBA1 gene therapy (Sanofi)AAV.GMU1-SS3-hGBA1
ClassificationCell and gene therapy > Gene therapy > Viral vector-based gene therapy > Adeno-associated virus vector
Mechanisms of ActionBeta-glucocerebrosidase stimulants
TargetsBeta-glucocerebrosidase
Therapy Area/IndicationsParkinson Disease
Global Approved IndicationsN/A
Approved Indications in ChinaN/A
Original Approved Country/TerritoryN/A
Original Approved DateN/A
Country/TerritoryUnited States
CombinationsNo
Global Highest StatusPreclinical
Highest Status in ChinaN/A
Originator CompaniesGenzyme Corp
China Registration ClassN/A

Develop Information

Active CompaniesGenzyme Corp
Inactive CompaniesN/A
Active IndicationsParkinson Disease
Inactive IndicationsN/A

Other Information

ATC CodeN/A
Related LinksN/A