Last update 01 Aug 2026

scAAV1.tMCK.NTF3(Nationwide Children's Hospital)

Overview

Basic Info

Drug Type
AAV based gene therapy
Synonyms
Neurotrophin 3 gene therapy, NT 3 gene therapy
Target
NTF3
Action-
Mechanism
Neurotrophin-3 expression stimulants, Gene transference(Gene transference)
Therapeutic Areas
Nervous System DiseasesCongenital DisordersOther Diseases
Active Indication-
Inactive Indication
Charcot-Marie-Tooth Disease, Type Ia
Originator Organization
Nationwide Children's HospitalNationwide Children's Hospital
Active Organization-
Inactive Organization
Nationwide Children's HospitalNationwide Children's Hospital
License Organization
Sarepta Therapeutics, Inc.Sarepta Therapeutics, Inc.Nationwide Children's HospitalNationwide Children's Hospital
Drug Highest PhaseDiscontinuedPhase 1/2
First Approval Date-
Regulation-
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Related

1
Clinical Trials associated with scAAV1.tMCK.NTF3(Nationwide Children's Hospital)
NCT03520751
/ WithdrawnPhase 1/2IIT
Phase I/IIa Trial Evaluating scAAV1.tMCK.NTF3 for Treatment of Charcot-Marie-Tooth Neuropathy Type 1A (CMT1A)
100 Clinical Results associated with scAAV1.tMCK.NTF3(Nationwide Children's Hospital)
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100 Translational Medicine associated with scAAV1.tMCK.NTF3(Nationwide Children's Hospital)
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100 Patents (Medical) associated with scAAV1.tMCK.NTF3(Nationwide Children's Hospital)
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100 Deals associated with scAAV1.tMCK.NTF3(Nationwide Children's Hospital)
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R&D Status

10 top R&D records.
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IndicationHighest PhaseCountry/LocationOrganizationDate
Charcot-Marie-Tooth Disease, Type IaPhase 2
United States
Nationwide Children's HospitalNationwide Children's Hospital
01 Apr 2027
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Clinical Result

Indication
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Approval

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Regulation

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