lentiviral vector gene CRISPR/Cas9 therapy (Rocket Pharmaceuticals/CIBERER)

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Report date: 2025/03/20 15:51:05

Base Information

Drug Namelentiviral vector gene CRISPR/Cas9 therapy (Rocket Pharmaceuticals/CIBERER)
Drug Alias NameN/A
ClassificationCell and gene therapy > Gene therapy > Viral vector-based gene therapy > Retroviral vectorCell and gene therapy > Gene therapy > Gene editing > CRISPR > CRISPR-Cas9
Mechanisms of ActionFanconi anemia group A protein modulators
TargetsFanconi anemia group A protein
Therapy Area/IndicationsFanconi Anemia
Global Approved IndicationsN/A
Approved Indications in ChinaN/A
Original Approved Country/TerritoryN/A
Original Approved DateN/A
Country/TerritoryUnited StatesSpain
CombinationsNo
Global Highest StatusPreclinical
Highest Status in ChinaN/A
Originator CompaniesRocket Pharma Ltd
China Registration ClassN/A

Develop Information

Active CompaniesCentre For Biomedical Network Research On Rare DiseasesRocket Pharmaceuticals Inc
Inactive CompaniesRocket Pharma Ltd
Active IndicationsFanconi Anemia
Inactive IndicationsN/A

Other Information

ATC CodeN/A
Related LinksN/A