PAH gene modulator LNP based AAV gene therapy (Tome Biosciences)

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Report date: 2025/06/11 10:53:45

Base Information

Drug NamePAH gene modulator LNP based AAV gene therapy (Tome Biosciences)

Drug Alias Nameintegrative gene therapy (Tome Biosciences)

ClassificationCell and gene therapy > Gene therapy > Viral vector-based gene therapy > Adeno-associated virus vectorSmall molecules > Nucleosides and nucleotide derivtives > OligonucleotideCell and gene therapy > Gene therapy > Gene editing > CRISPR > CRISPR-Cas9 +[4]

Mechanisms of ActionPhenylalanine hydroxylase modulators

TargetsPhenylalanine hydroxylase

Therapy Area/IndicationsPhenylketonurias

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 CompaniesTome Biosciences Inc

China Registration ClassN/A

Develop Information

Active CompaniesTome Biosciences Inc

Inactive CompaniesN/A

Active IndicationsPhenylketonurias

Inactive IndicationsN/A

Other Information

ATC CodeN/A