ex vivo gene-transduced autologous hematopoietic stem cell-based gene therapy (Altheia science/University of Padova/Dana-Farber/Boston Children's Cancer and Blood Disorders Center)

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Report date: 2025/06/11 10:34:52

Base Information

Drug Nameex vivo gene-transduced autologous hematopoietic stem cell-based gene therapy (Altheia science/University of Padova/Dana-Farber/Boston Children's Cancer and Blood Disorders Center)
Drug Alias NameIDS gene therapy (Altheia science/University of Padova/Dana-Farber/Boston Children's Cancer and Blood Disorders Center)
ClassificationCell and gene therapy > Cell therapy > Stem cell therapy > Hematopoietic stem cells therapyCell and gene therapy > Gene therapy > Viral vector-based gene therapy > Lentiviral vector
Mechanisms of ActionIduronate-2-sulfatase gene transference
TargetsIduronate-2-sulfatase
Therapy Area/IndicationsMucopolysaccharidosis II
Global Approved IndicationsN/A
Approved Indications in ChinaN/A
Original Approved Country/TerritoryN/A
Original Approved DateN/A
Country/TerritoryUnited StatesItaly
CombinationsNo
Global Highest StatusPreclinical
Highest Status in ChinaN/A
Originator CompaniesUniversity Of Padova
China Registration ClassN/A

Develop Information

Active CompaniesAltheia Science SrlDana-Farber/Boston Children's Cancer and Blood Disorders CenterUniversity Of Padova
Inactive CompaniesN/A
Active IndicationsMucopolysaccharidosis II
Inactive IndicationsN/A

Other Information

ATC CodeN/A
Related LinksN/A