Last update 08 May 2025

rAAV.TCAP

Overview

Basic Info

Drug Type
AAV based gene therapy
Synonyms
rAAV-Mediated TCAP Gene
Target
telethonin
Action
modulators
Mechanism
telethonin modulators(titin-cap modulators)
Therapeutic Areas
Nervous System DiseasesCongenital DisordersSkin and Musculoskeletal Diseases+ [1]
Active Indication
Muscular Dystrophy, Limb-Girdle, Type 2G
Inactive Indication-
Originator Organization
UMass Chan Medical SchoolUMass Chan Medical SchoolResearch Institute at Nationwide Childrens HospitalResearch Institute at Nationwide Childrens Hospital
Active Organization
UMass Chan Medical SchoolUMass Chan Medical SchoolResearch Institute at Nationwide Childrens HospitalResearch Institute at Nationwide Childrens Hospital
Inactive Organization-
License Organization-
Drug Highest PhasePreclinical
First Approval Date-
Regulation-

Related

100 Clinical Results associated with rAAV.TCAP
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100 Translational Medicine associated with rAAV.TCAP
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100 Patents (Medical) associated with rAAV.TCAP
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100 Deals associated with rAAV.TCAP
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R&D Status

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IndicationHighest PhaseCountry/LocationOrganizationDate
Muscular Dystrophy, Limb-Girdle, Type 2GPreclinical
United States
Research Institute at Nationwide Childrens HospitalResearch Institute at Nationwide Childrens Hospital
-
Muscular Dystrophy, Limb-Girdle, Type 2GPreclinical
United States
UMass Chan Medical SchoolUMass Chan Medical School
-
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Clinical Result

Indication
Phase
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Study
Phase
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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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