scAAV2/MFSD8 gene replacement therapy (Northwestern University Feinberg School of Medicine/UT Southwestern)

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

Base Information

Drug NamescAAV2/MFSD8 gene replacement therapy (Northwestern University Feinberg School of Medicine/UT Southwestern)
Drug Alias NameAAV9-based gene replacement therapy (Northwestern University Feinberg School of Medicine/UT Southwestern)AAV9/CLN7 (Northwestern University Feinberg School of Medicine/UT Southwestern)
ClassificationCell and gene therapy > Gene therapy > Viral vector-based gene therapy > Adeno-associated virus vector
Mechanisms of ActionUpdating
TargetsUpdating
Therapy Area/IndicationsLysosomal Storage DiseasesNeuronal Ceroid-Lipofuscinoses
Global Approved IndicationsN/A
Approved Indications in ChinaN/A
Original Approved Country/TerritoryN/A
Original Approved DateN/A
Country/TerritoryUnited States
CombinationsNo
Global Highest StatusPhase 1 Clinical
Highest Status in ChinaN/A
Originator CompaniesNorthwestern University Feinberg School Of Medicine
China Registration ClassN/A

Develop Information

Active CompaniesNorthwestern University Feinberg School Of MedicineUniversity of Texas Southwestern Medical Center
Inactive CompaniesN/A
Active IndicationsLysosomal Storage DiseasesNeuronal Ceroid-Lipofuscinoses
Inactive IndicationsN/A

Other Information

ATC CodeN/A
Related LinksN/A