adeno-associated viral vector delivered human glucocerebrosidase gene therapy (Royal Free Hospital)

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Report date: 2025/03/14 20:00:48

Base Information

Drug Nameadeno-associated viral vector delivered human glucocerebrosidase gene therapy (Royal Free Hospital)
Drug Alias NameAAV9-GBA gene therapy (Royal Free Hospital)
ClassificationCell and gene therapy > Gene therapy > Viral vector-based gene therapy > Adeno-associated virus vector
Mechanisms of ActionBeta-glucocerebrosidase stimulants
TargetsBeta-glucocerebrosidase
Therapy Area/IndicationsGaucher's disease type II
Global Approved IndicationsN/A
Approved Indications in ChinaN/A
Original Approved Country/TerritoryN/A
Original Approved DateN/A
Country/TerritoryUnited Kingdom
CombinationsNo
Global Highest StatusPending (Preclinical)
Highest Status in ChinaN/A
Originator CompaniesRoyal Free and University College Medical School
China Registration ClassN/A

Develop Information

Active CompaniesN/A
Inactive CompaniesRoyal Free and University College Medical School
Active IndicationsN/A
Inactive IndicationsGaucher's disease type II

Other Information

ATC CodeN/A
Related LinksN/A