Adeno-Associated Viral Vector Expressing Low-Density Lipoprotein Receptor

N/A

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

Base Information

Drug NameAdeno-Associated Viral Vector Expressing Low-Density Lipoprotein Receptor

Drug Alias NameRGX-501

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

Mechanisms of ActionLDLR gene transference

TargetsLDL receptor

Therapy Area/IndicationsHomozygous familial hypercholesterolemia

Global Approved IndicationsN/A

Approved Indications in ChinaN/A

Original Approved Country/TerritoryN/A

Original Approved DateN/A

Country/TerritoryCanadaUnited StatesNetherlands +[4]

CombinationsNo

Global Highest StatusDiscontinued (Phase 2 Clinical)

Highest Status in ChinaN/A

Originator CompaniesRegenxbio Inc

China Registration ClassN/A

Active CompaniesN/A

Inactive CompaniesRegenxbio Inc

Active IndicationsN/A

Inactive IndicationsHomozygous familial hypercholesterolemia

ATC CodeN/A