utrophin CRISPR/Cas9-gene therapy (University College London/Genethon)

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Report date: 2025/01/26 16:13:57

Base Information

Drug Nameutrophin CRISPR/Cas9-gene therapy (University College London/Genethon)
Drug Alias NameAAV9-SpCas9/rAAV9-gmLet7cAAV9-utrophin gene therapy (University College London/Genethon)CRISPR/Cas9-based therapeutics (University College London/Genethon)utrophin gene therapy (University College London/Genethon)
ClassificationBiologics
Mechanisms of ActionUtrophin activators
TargetsUtrophin
Therapy Area/IndicationsMuscular Dystrophy, Duchenne
Global Approved IndicationsN/A
Approved Indications in ChinaN/A
Original Approved Country/TerritoryN/A
Original Approved DateN/A
Country/TerritoryUnited KingdomFrance
CombinationsNo
Global Highest StatusPreclinical
Highest Status in ChinaN/A
Originator CompaniesUniversity College London
China Registration ClassN/A

Develop Information

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

Other Information

ATC CodeN/A
Related LinksN/A