adeno-associated virus serotype 9 vector-delivered SMN1 gene therapy(Genzyme/University of California San Francisco/Emory University/Georgetown University)

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Report date: 2025/02/01 20:00:06

Base Information

Drug Nameadeno-associated virus serotype 9 vector-delivered SMN1 gene therapy(Genzyme/University of California San Francisco/Emory University/Georgetown University)
Drug Alias NameAAV2/8-CBA-hSMN1SMN1 gene therapy (AAV8-serotype vector) (Genzyme)scAAV2/8-GUSB-hSMN1scAAV9-hSMN1survival motor neuron 1 gene therapy (adeno-associated viral vector) (Genzyme)
ClassificationBiologics
Mechanisms of ActionSurvival motor neuron protein stimulants
TargetsSurvival motor neuron protein
Therapy Area/IndicationsMuscular Atrophy, Spinal
Global Approved IndicationsN/A
Approved Indications in ChinaN/A
Original Approved Country/TerritoryN/A
Original Approved DateN/A
Country/TerritoryUnited States
CombinationsNo
Global Highest StatusPending (Preclinical)
Highest Status in ChinaN/A
Originator CompaniesGenzyme Corp
China Registration ClassN/A

Develop Information

Active CompaniesN/A
Inactive CompaniesUniversity Of California San FranciscoGeorgetown UniversityGenzyme Corp +[4] 
Active IndicationsN/A
Inactive IndicationsMuscular Atrophy, Spinal

Other Information

ATC CodeN/A
Related LinksN/A