Last update 08 May 2025

ADA lentiviral vector-transduced hematopoietic stem cell gene therapy(Fondazione Telethon)

Overview

Basic Info

Drug Type
Gene therapy, Hematopoietic stem cell therapy
Synonyms
lentiviral vector (LV) mediated hematopoietic stem progenitor cell (HSPCs) gene therapy
Target-
Action-
Mechanism
Stem cell replacements
Therapeutic Areas
Immune System DiseasesCongenital DisordersEndocrinology and Metabolic Disease+ [2]
Active Indication-
Inactive Indication
Adenosine deaminase deficiency
Originator Organization
Fondazione Telethon EtsFondazione Telethon Ets
Active Organization-
Inactive Organization
Fondazione Telethon EtsFondazione Telethon Ets
License Organization-
Drug Highest PhasePendingDiscovery
First Approval Date-
RegulationOrphan Drug (European Union)

Related

100 Clinical Results associated with ADA lentiviral vector-transduced hematopoietic stem cell gene therapy(Fondazione Telethon)
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100 Translational Medicine associated with ADA lentiviral vector-transduced hematopoietic stem cell gene therapy(Fondazione Telethon)
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100 Patents (Medical) associated with ADA lentiviral vector-transduced hematopoietic stem cell gene therapy(Fondazione Telethon)
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100 Deals associated with ADA lentiviral vector-transduced hematopoietic stem cell gene therapy(Fondazione Telethon)
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R&D Status

10 top R&D records.
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IndicationHighest PhaseCountry/LocationOrganizationDate
Adenosine deaminase deficiencyDiscovery
Italy
Fondazione Telethon EtsFondazione Telethon Ets
16 Nov 2020
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Clinical Result

Indication
Phase
Evaluation
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Study
Phase
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Translational Medicine

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Deal

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Core Patent

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Clinical Trial

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Approval

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Regulation

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