GEN-6050X

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Report date: 2025/09/18 15:54:09

Base Information

Drug NameGEN-6050X
Drug Alias NameExon 50 skipping DMD therapeutic (Genassist Therapeutics)Exon 50 skipping RNA (Genassist Therapeutics)GEN-6050GEN-6050XGEN6050X
ClassificationCell and gene therapy > Gene therapy > Gene editing > Base editingCell and gene therapy > Gene therapy > Viral vector-based gene therapy > Adeno-associated virus vector
Mechanisms of ActionGene expression modulatorsDystrophin-associated proteins stimulants
TargetsDystrophin-associated proteins
Therapy Area/IndicationsMuscular Dystrophy, DuchenneMuscular Dystrophies
Global Approved IndicationsN/A
Approved Indications in ChinaN/A
Original Approved Country/TerritoryN/A
Original Approved DateN/A
Country/TerritoryUnited StatesMainland China
CombinationsNo
Global Highest StatusPhase 1 Clinical
Highest Status in ChinaPhase 1 Clinical
Originator CompaniesSuzhou Genassist Therapeutics Co Ltd
China Registration ClassN/A

Develop Information

Active CompaniesSuzhou Genassist Therapeutics Co LtdPeking Union Medical College Hospital
Inactive CompaniesN/A
Active IndicationsMuscular Dystrophy, DuchenneMuscular Dystrophies
Inactive IndicationsN/A

Other Information

ATC CodeN/A
Related LinksN/A