Last update 21 Nov 2024

AAV gene therapy (Sarepta/Columbia University)

Overview

Basic Info

Drug Type
AAV based gene therapy
Synonyms-
Target-
Mechanism
Gene transference
Therapeutic Areas
Nervous System DiseasesCongenital DisordersSkin and Musculoskeletal Diseases+ [1]
Active Indication
Muscular Dystrophy, Emery-Dreifuss
Inactive Indication-
Originator Organization
Sarepta Therapeutics, Inc.Sarepta Therapeutics, Inc.
Active Organization
Sarepta Therapeutics, Inc.Sarepta Therapeutics, Inc.The Trustees of Columbia University in The City of New YorkThe Trustees of Columbia University in The City of New York
Inactive Organization
StrideBio, Inc.StrideBio, Inc.Startup
Drug Highest PhaseDiscovery
First Approval Date-
Regulation-

Related

100 Clinical Results associated with AAV gene therapy (Sarepta/Columbia University)
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100 Translational Medicine associated with AAV gene therapy (Sarepta/Columbia University)
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100 Patents (Medical) associated with AAV gene therapy (Sarepta/Columbia University)
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100 Deals associated with AAV gene therapy (Sarepta/Columbia University)
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R&D Status

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IndicationHighest PhaseCountry/LocationOrganizationDate
Muscular Dystrophy, Emery-DreifussPhase 2
US
StrideBio, Inc.StrideBio, Inc.Startup
-
Muscular Dystrophy, Emery-DreifussDiscovery
US
The Trustees of Columbia University in The City of New YorkThe Trustees of Columbia University in The City of New York
-
Muscular Dystrophy, Emery-DreifussDiscovery
US
Sarepta Therapeutics, Inc.Sarepta Therapeutics, Inc.
-
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Clinical Result

Indication
Phase
Evaluation
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Study
Phase
PopulationAnalyzed EnrollmentGroupResultsEvaluationPublication Date

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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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