anti-TfR1 ASM gene therapy (AAV9, Regeneron Pharmaceuticals)

N/A

Report date: 2026/05/20 20:02:01

Base Information

Drug Nameanti-TfR1 ASM gene therapy (AAV9, Regeneron Pharmaceuticals)

Drug Alias Nameanti-TfR1:AAV9:ASM

ClassificationBiologics > Protein drugs > Immunoglobulin > Antibody > Antibody fragmentCell and gene therapy > Gene therapy > Viral vector-based gene therapy > Adeno-associated virus vector

Mechanisms of ActionTransferrin receptor protein 1 modulatorsSphingomyelin phosphodiesterase stimulants

TargetsTransferrin receptor protein 1Sphingomyelin phosphodiesterase

Therapy Area/IndicationsNiemann-Pick Diseases

Global Approved IndicationsN/A

Approved Indications in ChinaN/A

Original Approved Country/TerritoryN/A

Original Approved DateN/A

Country/TerritoryUnited States

CombinationsNo

Global Highest StatusPreclinical

Highest Status in ChinaN/A

Originator CompaniesRegeneron Pharmaceuticals Inc

China Registration ClassN/A

Develop Information

Active CompaniesRegeneron Pharmaceuticals Inc

Inactive CompaniesN/A

Active IndicationsNiemann-Pick Diseases

Inactive IndicationsN/A

Other Information

ATC CodeN/A