dystrophin exon-skipping gene therapy (Genethon)

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

Base Information

Drug Namedystrophin exon-skipping gene therapy (Genethon)
Drug Alias NameAAV1-U7 gene therapy (Genethon)rAAV2/8 U7 RNAOpt ESE6-ESE8dystrophin gene therapy (Genethon)
ClassificationSmall molecules > Nucleosides and nucleotide derivtives > Oligonucleotide > Oligonucleotide antisense
Mechanisms of ActionDystrophin gene transference
TargetsDystrophin
Therapy Area/IndicationsMuscular Dystrophy, Duchenne
Global Approved IndicationsN/A
Approved Indications in ChinaN/A
Original Approved Country/TerritoryN/A
Original Approved DateN/A
Country/TerritoryWestern Europe
CombinationsNo
Global Highest StatusPending (Preclinical)
Highest Status in ChinaN/A
Originator CompaniesGenethon
China Registration ClassN/A

Develop Information

Active CompaniesN/A
Inactive CompaniesGenethon
Active IndicationsN/A
Inactive IndicationsMuscular Dystrophy, Duchenne

Other Information

ATC CodeN/A
Related LinksN/A