ADA lentiviral vector-transduced hematopoietic stem cell gene therapy (Orchard Therapeutics/San Raffaele-Telethon Institute for Gene Therapy)

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Report date: 2025/03/14 20:00:48

Base Information

Drug NameADA lentiviral vector-transduced hematopoietic stem cell gene therapy (Orchard Therapeutics/San Raffaele-Telethon Institute for Gene Therapy)
Drug Alias Namelentiviral vector mediated hematopoietic stem progenitor cell gene therapy (Orchard Therapeutics/San Raffaele-Telethon Institute for Gene Therapy)
ClassificationCell 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 ActionADA gene transference
TargetsAdenosine deaminase
Therapy Area/IndicationsAdenosine deaminase deficiency
Global Approved IndicationsN/A
Approved Indications in ChinaN/A
Original Approved Country/TerritoryN/A
Original Approved DateN/A
Country/TerritoryUnited Kingdom
CombinationsNo
Global Highest StatusClinical
Highest Status in ChinaN/A
Originator CompaniesOrchard Therapeutics Plc
China Registration ClassN/A

Develop Information

Active CompaniesThe San Raffaele Telethon Institute For Gene TherapyOrchard Therapeutics Plc
Inactive CompaniesN/A
Active IndicationsAdenosine deaminase deficiency
Inactive IndicationsN/A

Other Information

ATC CodeN/A
Related LinksN/A