Autologous CD34+ cells transduced with a lentiviral vector containing the human SGSH gene (Orchard)

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Report date: 2023/10/26 16:20:49

Base Information

Drug NameAutologous CD34+ cells transduced with a lentiviral vector containing the human SGSH gene (Orchard)
Drug Alias NameOTL-201
ClassificationCell and gene therapy > Cell therapy > Donor > AutologousCell and gene therapy > Gene therapy > Viral vector-based gene therapy > Lentiviral vectorCell and gene therapy > Cell therapy > Stem cell therapy > Hematopoietic stem cells therapy
Mechanisms of ActionCell replacementsSGSH gene transference
TargetsN-sulphoglucosamine sulphohydrolase
Therapy Area/IndicationsMucopolysaccharidosis III
Global Approved IndicationsN/A
Approved Indications in ChinaN/A
Original Approved Country/TerritoryN/A
Original Approved DateN/A
Country/TerritoryUnited Kingdom
CombinationsNo
Global Highest StatusPhase 2 Clinical
Highest Status in ChinaN/A
Originator CompaniesOrchard Therapeutics Plc
China Registration ClassN/A

Develop Information

Active CompaniesOrchard Therapeutics PlcThe University Of Manchester
Inactive CompaniesN/A
Active IndicationsMucopolysaccharidosis III
Inactive IndicationsN/A

Other Information

ATC CodeN/A
Related LinksN/A