AVXS-301

N/A

Report date: 2025/01/26 18:07:39

NME

Base Information

Drug NameAVXS-301

Drug Alias NameAAV9-SOD1-shRNA (AveXis)NAV rAAV9 vector (AveXis)superoxide dismutase 1 gene silencing therapy (AveXis)adeno-associated virus serotype 9 vector-delivered SOD1 gene therapy (AveXis)AVXS-301

ClassificationSmall molecules > Nucleosides and nucleotide derivtives > Oligonucleotide > short hairpin RNA

Mechanisms of ActionSuperoxide dismutase inhibitors

TargetsSuperoxide dismutase

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 StatesEurope

CombinationsNo

Global Highest StatusPreclinical

Highest Status in ChinaN/A

Originator CompaniesNovartis Gene Therapies Inc

China Registration ClassN/A

Develop Information

Active CompaniesNovartis Gene Therapies Inc

Inactive CompaniesN/A

Active IndicationsAmyotrophic Lateral Sclerosis

Inactive IndicationsN/A

Other Information

ATC CodeN/A