AAV-delivered CRISPR/Cas9 NRL-silencing gene therapy (National Eye Institute)

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Report date: 2025/03/14 16:46:51

Base Information

Drug NameAAV-delivered CRISPR/Cas9 NRL-silencing gene therapy (National Eye Institute)

Drug Alias NameNRL-silencing gene therapy (National Eye Institute)

ClassificationCell and gene therapy > Gene therapy > Viral vector-based gene therapy > Adeno-associated virus vectorCell and gene therapy > Gene therapy > Gene editing > CRISPR > CRISPR-Cas9

Mechanisms of ActionTranscription factor Maf inhibitors

TargetsTranscription factor Maf

Therapy Area/IndicationsRetinitis Pigmentosa

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 CompaniesNational Eye Institute

China Registration ClassN/A

Develop Information

Active CompaniesNational Eye Institute

Inactive CompaniesN/A

Active IndicationsRetinitis Pigmentosa

Inactive IndicationsN/A

Other Information

ATC CodeN/A