PM-AAV9-miAtxn2

N/A

Report date: 2026/04/10 20:01:49

Base Information

Drug NamePM-AAV9-miAtxn2

Drug Alias NameN/A

ClassificationSmall molecules > Nucleosides and nucleotide derivtives > Oligonucleotide > microRNACell and gene therapy > Gene therapy > Viral vector-based gene therapy > Adeno-associated virus vector > AAV9 vector

Mechanisms of ActionAtaxin-2 modulatorsGene silencing

TargetsAtaxin-2

Therapy Area/IndicationsAmyotrophic Lateral Sclerosis

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 CompaniesUniversity Of Pennsylvania

China Registration ClassN/A

Develop Information

Active CompaniesChildrens Hospital of Philadelphia University Of Pennsylvania

Inactive CompaniesN/A

Active IndicationsAmyotrophic Lateral Sclerosis

Inactive IndicationsN/A

Other Information

ATC CodeN/A