AAV9-vector based Gys1 gene therapy (Genethon/Inserm/Spark Therapeutics)

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Report date: 2026/01/27 17:57:26

Base Information

Drug NameAAV9-vector based Gys1 gene therapy (Genethon/Inserm/Spark Therapeutics)
Drug Alias NameCRISPR/Cas9-based therapeutics (Genethon/Inserm/Spark Therapeutics)Glycogen synthase CRISPR/Cas9-gene therapy(Genethon/Inserm/Spark Therapeutics)
ClassificationCell and gene therapy > Gene therapy > Viral vector-based gene therapy > Adeno-associated virus vector
Mechanisms of ActionGlycogen synthase 1 inhibitors
TargetsGlycogen synthase 1
Therapy Area/IndicationsGlycogen Storage Disease Type IIIGlycogen Storage DiseaseGlycogen Storage Disease Type II
Global Approved IndicationsN/A
Approved Indications in ChinaN/A
Original Approved Country/TerritoryN/A
Original Approved DateN/A
Country/TerritoryFrance
CombinationsNo
Global Highest StatusPreclinical
Highest Status in ChinaN/A
Originator CompaniesInstitut National De La Sante Et De La Recherche Medicale
China Registration ClassN/A

Develop Information

Active CompaniesInstitut National De La Sante Et De La Recherche MedicaleGenethonSpark Therapeutics Inc
Inactive CompaniesN/A
Active IndicationsGlycogen Storage Disease Type IIIGlycogen Storage DiseaseGlycogen Storage Disease Type II
Inactive IndicationsN/A

Other Information

ATC CodeN/A
Related LinksN/A