GAA gene therapy (Sarepta Therapeutics/Lacerta Therapeutics)

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Report date: 2025/01/15 13:53:47

Base Information

Drug NameGAA gene therapy (Sarepta Therapeutics/Lacerta Therapeutics)
Drug Alias NameN/A
ClassificationCell and gene therapy > Gene therapy
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 StatusPreclinical
Highest Status in ChinaN/A
Originator CompaniesSarepta Therapeutics Inc
China Registration ClassN/A

Develop Information

Active CompaniesLacerta Therapeutics IncSarepta Therapeutics Inc
Inactive CompaniesN/A
Active IndicationsGlycogen Storage Disease Type II
Inactive IndicationsN/A

Other Information

ATC CodeN/A
Related LinksN/A