Last update 20 Mar 2025

SMN2 gene therapy (UniQure)

Overview

Basic Info

Drug Type
AAV based gene therapy
Synonyms-
Target
SMN2
Action
modulators
Mechanism
SMN2 modulators(Survival motor neuron protein modulators), Gene transference
Therapeutic Areas
Nervous System DiseasesOther Diseases
Active Indication
Spinal Muscular Atrophy
Inactive Indication-
Originator Organization
uniQure NVuniQure NV
Active Organization
uniQure NVuniQure NVUniversity of FerraraUniversity of FerraraICGEBICGEB
+ [1]
Inactive Organization-
Drug Highest PhasePreclinical
First Approval Date-
Regulation-

Related

100 Clinical Results associated with SMN2 gene therapy (UniQure)
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100 Translational Medicine associated with SMN2 gene therapy (UniQure)
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100 Patents (Medical) associated with SMN2 gene therapy (UniQure)
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100 Deals associated with SMN2 gene therapy (UniQure)
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R&D Status

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IndicationHighest PhaseCountry/LocationOrganizationDate
Spinal Muscular AtrophyPreclinical
Italy
University of FerraraUniversity of Ferrara
-
Spinal Muscular AtrophyPreclinical
Italy
ICGEBICGEB
-
Spinal Muscular AtrophyPreclinical
Switzerland
University of BaselUniversity of Basel
-
Spinal Muscular AtrophyPreclinical
Netherlands
uniQure NVuniQure NV
-
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Clinical Result

Indication
Phase
Evaluation
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Phase
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Deal

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Clinical Trial

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Approval

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Regulation

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