Last update 21 Nov 2024
Human iduronate 2-sulfatase stem cell-based gene therapy(University of Padua)
Last update 21 Nov 2024
Overview
Basic Info
Drug Type Hematopoietic stem cell therapy |
Synonyms IDS-transduced autologous CD34-positive hematopoietic stem cell therapy, human IDS stem cell-based gene therapy, human iduronate 2-sulfatase gene therapy |
Target- |
Mechanism Stem cell replacements |
Therapeutic Areas |
Active Indication- |
Inactive Indication |
Originator Organization |
Active Organization- |
Inactive Organization |
Drug Highest PhasePendingDiscovery |
First Approval Date- |
RegulationOrphan Drug (EU) |
R&D Status
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| Indication | Highest Phase | Country/Location | Organization | Date |
|---|---|---|---|---|
| Mucopolysaccharidosis II | Discovery | IT | 15 Jul 2021 |
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Clinical Result
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