liver-targeted gene therapy (Sangamo Therapeutics/Icahn School of Medicine at Mount Sinai)

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Report date: 2025/03/20 20:00:06

Base Information

Drug Nameliver-targeted gene therapy (Sangamo Therapeutics/Icahn School of Medicine at Mount Sinai)
Drug Alias NameAAV encoding human GalA gene (Sangamo/Mount Sinai Med Cent)ZFN-mediated genome editing (Sangamo/Mount Sinai Med Cent)
ClassificationCell and gene therapy > Gene therapy > Viral vector-based gene therapy > Adeno-associated virus vector
Mechanisms of ActionUpdating
TargetsUpdating
Therapy Area/IndicationsFabry Disease
Global Approved IndicationsN/A
Approved Indications in ChinaN/A
Original Approved Country/TerritoryN/A
Original Approved DateN/A
Country/TerritoryUnited States
CombinationsNo
Global Highest StatusPending (Preclinical)
Highest Status in ChinaN/A
Originator CompaniesSangamo Therapeutics Inc
China Registration ClassN/A

Develop Information

Active CompaniesN/A
Inactive CompaniesSangamo Therapeutics IncIcahn School Of Medicine At Mount Sinai
Active IndicationsN/A
Inactive IndicationsFabry Disease

Other Information

ATC CodeN/A
Related LinksN/A