SMN2 gene therapy (UniQure)

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Report date: 2025/01/26 16:13:18

Base Information

Drug NameSMN2 gene therapy (UniQure)
Drug Alias NameAAV9-Exon Specific U1 snRNA (UniQure/ICGEB/Biozentrum Univ of Basel/Univ of Ferrara)SMN2 gene therapy (UniQure/ICGEB/Biozentrum Univ of Basel/Univ of Ferrara)
ClassificationBiologics
Mechanisms of ActionSMN2 gene modulators
TargetsUpdating
Therapy Area/IndicationsMuscular Atrophy, Spinal
Global Approved IndicationsN/A
Approved Indications in ChinaN/A
Original Approved Country/TerritoryN/A
Original Approved DateN/A
Country/TerritoryNetherlandsItalySwitzerland
CombinationsNo
Global Highest StatusPreclinical
Highest Status in ChinaN/A
Originator CompaniesUniqure Nv
China Registration ClassN/A

Develop Information

Active CompaniesUniqure NvUniversity of FerraraBiozentrum University of Basel +[4] 
Inactive CompaniesN/A
Active IndicationsMuscular Atrophy, Spinal
Inactive IndicationsN/A

Other Information

ATC CodeN/A
Related LinksN/A