AAV9-CaMKII-NPC1 (NHGRI)

N/A

Report date: 2025/01/26 18:11:28

Base Information

Drug NameAAV9-CaMKII-NPC1 (NHGRI)

Drug Alias NameAAV9-delivered NPC1 gene therapy (National Human Genome Research Institute)PHP.B-EF1a-NPC1 (NHGRI)AAV9-EF1a-NPC1 (National Human Genome Research Institute)

ClassificationBiologics

Mechanisms of ActionNPC Intracellular Cholesterol Transporter 1 stimulants

TargetsNPC Intracellular Cholesterol Transporter 1

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 CompaniesNational Human Genome Research Institute

China Registration ClassN/A

Develop Information

Active CompaniesNational Human Genome Research Institute

Inactive CompaniesN/A

Active IndicationsNiemann-Pick Diseases

Inactive IndicationsN/A

Other Information

ATC CodeN/A