CRX gene therapy (National Eye Institute)

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Report date: 2025/03/19 09:00:53

Base Information

Drug NameCRX gene therapy (National Eye Institute)
Drug Alias NameAAV-based CRX gene therapy (National Eye Institute/PTC Therapeutics)
ClassificationCell and gene therapy > Gene therapy > Viral vector-based gene therapy > Adeno-associated virus vector
Mechanisms of ActionCone-rod homeobox protein gene transference
TargetsCone-rod homeobox protein
Therapy Area/IndicationsLeber Congenital Amaurosis
Global Approved IndicationsN/A
Approved Indications in ChinaN/A
Original Approved Country/TerritoryN/A
Original Approved DateN/A
Country/TerritoryUnited States
CombinationsNo
Global Highest StatusPreclinical
Highest Status in ChinaN/A
Originator CompaniesPtc Therapeutics Inc
China Registration ClassN/A

Develop Information

Active CompaniesNational Eye InstitutePtc Therapeutics Inc
Inactive CompaniesN/A
Active IndicationsLeber Congenital Amaurosis
Inactive IndicationsN/A

Other Information

ATC CodeN/A
Related LinksN/A