RGX-381

N/A

Report date: 2026/01/20 17:48:50

Base Information

Drug NameRGX-381
Drug Alias NameAAV9.CB7.hCLN2RGX-381TTX-381
ClassificationCell and gene therapy > Gene therapy > Viral vector-based gene therapy > Adeno-associated virus vector > AAV9 vector
Mechanisms of ActionGene transferenceTripeptidyl peptidase 1 replacements
TargetsTripeptidyl Peptidase 1
Therapy Area/IndicationsNeuronal ceroid lipofuscinosis type 2Neuronal Ceroid-Lipofuscinoses
Global Approved IndicationsN/A
Approved Indications in ChinaN/A
Original Approved Country/TerritoryN/A
Original Approved DateN/A
Country/TerritoryGermanyUnited Kingdom
CombinationsNo
Global Highest StatusPhase 2 Clinical
Highest Status in ChinaN/A
Originator CompaniesRegenxbio Inc
China Registration ClassN/A

Develop Information

Active CompaniesTern TherapeuticsRegenxbio Inc
Inactive CompaniesN/A
Active IndicationsNeuronal ceroid lipofuscinosis type 2Neuronal Ceroid-Lipofuscinoses
Inactive IndicationsN/A

Other Information

ATC CodeN/A
Related LinksN/A