phenylalanine-4-hydroxylase gene therapy (Generation Bio)

N/A

Report date: 2025/01/26 17:44:57

NME

Base Information

Drug Namephenylalanine-4-hydroxylase gene therapy (Generation Bio)

Drug Alias NameceDNA construct gene therapy (Generation Bio)closed-ended DNA construct gene therapy (Generation Bio)PAH gene stimulator (Generation Bio)PAH gene therapy (Generation Bio)

ClassificationSmall molecules > Nucleosides and nucleotide derivtives > Oligonucleotide

Mechanisms of ActionPhenylalanine hydroxylase stimulants

TargetsPhenylalanine hydroxylase

Therapy Area/IndicationsMetabolic DiseasesPhenylketonurias

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 CompaniesGeneration Bio Co

China Registration ClassN/A

Develop Information

Active CompaniesGeneration Bio Co

Inactive CompaniesN/A

Active IndicationsPhenylketonurias

Inactive IndicationsMetabolic Diseases

Other Information

ATC CodeN/A