UBE3A gene therapy (HuidaGene Therapeutics/Chinese Academy of Sciences)

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Report date: 2025/06/11 11:02:23

Base Information

Drug NameUBE3A gene therapy (HuidaGene Therapeutics/Chinese Academy of Sciences)
Drug Alias NameAAV-hfCax12Max-UBE3A gRNAAAV-hfCas13Y-UBE3A gRNA
ClassificationCell and gene therapy > Gene therapy > Viral vector-based gene therapy > Adeno-associated virus vectorCell and gene therapy > Gene therapy > Gene editing > CRISPR > CRISPR-Cas13
Mechanisms of ActionUbiquitin-like modifier-activating enzyme 3A modulatorsNeuroprotectants
TargetsUbiquitin-like modifier-activating enzyme 3A
Therapy Area/IndicationsAngelman Syndrome
Global Approved IndicationsN/A
Approved Indications in ChinaN/A
Original Approved Country/TerritoryN/A
Original Approved DateN/A
Country/TerritoryMainland China
CombinationsNo
Global Highest StatusPreclinical
Highest Status in ChinaPreclinical
Originator CompaniesChinese Academy Of Sciences
China Registration ClassN/A

Develop Information

Active CompaniesHuidaGene Therapeutics Co LtdChinese Academy Of Sciences
Inactive CompaniesN/A
Active IndicationsAngelman Syndrome
Inactive IndicationsN/A

Other Information

ATC CodeN/A
Related LinksN/A