RP-A501

N/A

Report date: 2025/05/29 14:08:06

Base Information

Drug NameRP-A501
Drug Alias NameRP-A501RP-A501 (rAAV9 capsid containing the human LAMP2B transgene)
ClassificationCell and gene therapy > Gene therapy > Viral vector-based gene therapy > Adeno-associated virus vector > AAV9 vector
Mechanisms of ActionGene transferenceLysosome-associated membrane protein 2 stimulants
TargetsLysosome-associated membrane protein 2
Therapy Area/IndicationsGlycogen Storage Disease Type IIb
Global Approved IndicationsN/A
Approved Indications in ChinaN/A
Original Approved Country/TerritoryN/A
Original Approved DateN/A
Country/TerritoryUnited StatesItalyGermany +[4] 
CombinationsNo
Global Highest StatusPhase 2 Clinical
Highest Status in ChinaN/A
Originator CompaniesRocket Pharmaceuticals Inc
China Registration ClassN/A

Develop Information

Active CompaniesRocket Pharmaceuticals Inc
Inactive CompaniesN/A
Active IndicationsGlycogen Storage Disease Type IIb
Inactive IndicationsN/A

Other Information

ATC CodeN/A
Related LinksN/A