UBE3A AAV gene therapies

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Report date: 2023/10/26 17:02:59

Base Information

Drug NameUBE3A AAV gene therapies
Drug Alias Namerecombinant adeno-associated virus serotype 9 vector containing the transgene UBE3A encoding for ubiquitin protein ligase E3A/E6-AP；Recombinant adeno-associated virus vector containing the active biological substance AAV-GTX-hUBE3AGT-AS
ClassificationCell and gene therapy > Gene therapy > Viral vector-based gene therapy > Adeno-associated virus vector
Mechanisms of ActionUbiquitin-like modifier-activating enzyme 3A stimulants
TargetsUbiquitin-like modifier-activating enzyme 3A
Therapy Area/IndicationsFragile X SyndromeAngelman Syndrome
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 CompaniesPtc Therapeutics IncAgilis Biotherapeutics
China Registration ClassN/A

Develop Information

Active CompaniesAgilis Biotherapeutics
Inactive CompaniesN/A
Active IndicationsFragile X SyndromeAngelman Syndrome
Inactive IndicationsN/A

Other Information

ATC CodeN/A
Related LinksN/A