PBML-04

N/A

Report date: 2023/10/26 16:53:40

Base Information

Drug NamePBML-04
Drug Alias NamePBML-04
ClassificationCell and gene therapy > Gene therapy > Viral vector-based gene therapy > Adeno-associated virus vector
Mechanisms of ActionARSA gene transference
TargetsCerebroside-sulfatase
Therapy Area/IndicationsLeukodystrophy, Metachromatic
Global Approved IndicationsN/A
Approved Indications in ChinaN/A
Original Approved Country/TerritoryN/A
Original Approved DateN/A
Country/TerritoryUnited States
CombinationsNo
Global Highest StatusPhase 1 Clinical
Highest Status in ChinaN/A
Originator CompaniesUniversity Of Pennsylvania
China Registration ClassN/A

Develop Information

Active CompaniesPassage Bio IncGemma Biotherapeutics Inc
Inactive CompaniesN/A
Active IndicationsLeukodystrophy, Metachromatic
Inactive IndicationsN/A

Other Information

ATC CodeN/A
Related LinksN/A