AAV9-CLN3 (Amicus Therapeutics)

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Report date: 2023/10/26 16:15:00

Base Information

Drug NameAAV9-CLN3 (Amicus Therapeutics)
Drug Alias NameCLN3-Batten diseaseCLN3 gene therapyAAV-CLN3-Amicus TherapeuticsAAV9-CLN3AT-GTX-502
ClassificationCell and gene therapy > Gene therapy > Viral vector-based gene therapy > Adeno-associated virus vector > AAV9 vector
Mechanisms of ActionCLN3 expression stimulantsGene transference
TargetsBattenin
Therapy Area/IndicationsNeuronal Ceroid-Lipofuscinoses
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 CompaniesNationwide Children'S HospitalThe Ohio State University
China Registration ClassN/A

Develop Information

Active CompaniesAlcyone Therapeutics Inc
Inactive CompaniesN/A
Active IndicationsNeuronal Ceroid-Lipofuscinoses
Inactive IndicationsN/A

Other Information

ATC CodeN/A
Related LinksN/A