Last update 08 May 2025

Hereditary inclusion body myopathy gene therapy(Gradalis, Inc.)

Overview

Basic Info

Drug Type
Gene therapy
Synonyms
GNE Lipoplex, Hereditary inclusion body myopathy gene therapy
Target
GNE
Action-
Mechanism
GNE gene transference
Therapeutic Areas
Nervous System DiseasesCongenital DisordersSkin and Musculoskeletal Diseases+ [1]
Active Indication-
Inactive Indication
Distal MyopathiesDistal Myopathy, Nonaka Type
Originator Organization
Gradalis, Inc.Gradalis, Inc.
Active Organization-
Inactive Organization
Gradalis, Inc.Gradalis, Inc.
License Organization-
Drug Highest PhasePendingPhase 1
First Approval Date-
RegulationOrphan Drug (United States)
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Related

100 Clinical Results associated with Hereditary inclusion body myopathy gene therapy(Gradalis, Inc.)
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100 Translational Medicine associated with Hereditary inclusion body myopathy gene therapy(Gradalis, Inc.)
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100 Patents (Medical) associated with Hereditary inclusion body myopathy gene therapy(Gradalis, Inc.)
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100 Deals associated with Hereditary inclusion body myopathy gene therapy(Gradalis, Inc.)
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R&D Status

10 top R&D records.
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IndicationHighest PhaseCountry/LocationOrganizationDate
Distal MyopathiesPhase 1-
Gradalis, Inc.Gradalis, Inc.
-
Distal Myopathy, Nonaka TypePhase 1
United States
Gradalis, Inc.Gradalis, Inc.
-
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Clinical Result

Indication
Phase
Evaluation
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Study
Phase
PopulationAnalyzed EnrollmentGroupResultsEvaluationPublication Date
ASGCT2010
Not Applicable
Distal Myopathy, Nonaka Type
-
wvxylihcoo(vthsoqhvji) = xcejskwryc xzqlbjdknw (gqdhgmbrur )
-
01 May 2010
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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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