RGNX-003

N/A

Report date: 2025/06/11 20:01:15

Base Information

Drug NameRGNX-003

Drug Alias NameArylsulfatase B Gene Modulator (RegenX Biosciences)Maroteaux-Lamy syndrome gene therapy (RegeneX Biosciences)adeno-associated virus vector-8 based gene therapy (RegenX Biosiences)MPS-AAV8RGNX-003

ClassificationCell and gene therapy > Gene therapy > Viral vector-based gene therapy > Adeno-associated virus vector

Mechanisms of ActionArylsulfatase B stimulants

TargetsArylsulfatase B

Therapy Area/IndicationsMucopolysaccharidosis VI

Global Approved IndicationsN/A

Approved Indications in ChinaN/A

Original Approved Country/TerritoryN/A

Original Approved DateN/A

Country/TerritoryUnited States

CombinationsNo

Global Highest StatusPending (Preclinical)

Highest Status in ChinaN/A

Originator CompaniesRegenxbio Inc

China Registration ClassN/A

Develop Information

Active CompaniesN/A

Inactive CompaniesRegenxbio Inc

Active IndicationsN/A

Inactive IndicationsMucopolysaccharidosis VI

Other Information

ATC CodeN/A