Last update 23 Jan 2025

AAV1-gamma-sarcoglycan vector(Viromed, Inc.)

Overview

Basic Info

Drug Type
AAV based gene therapy
Synonyms
AAV1-gamma-sarcoglycan gene therapy - Genethon, AAV1-gamma-sarcoglycan vector injection - Genethon, Adeno-associated viral 1 vector-mediated gamma-sarcoglycan gene therapy
+ [2]
Target
SGCG
Mechanism
Gamma-sarcoglycan gene transference
Therapeutic Areas
Nervous System DiseasesCongenital DisordersSkin and Musculoskeletal Diseases+ [1]
Active Indication
Muscular Dystrophies, Limb-Girdle
Inactive Indication
Limb-Girdle Muscular Dystrophy, Type 2CSarcoglycanopathies
Originator Organization
Viromed, Inc.Viromed, Inc.
Active Organization
Viromed, Inc.Viromed, Inc.GenethonGenethon
Inactive Organization-
Drug Highest PhasePhase 1
First Approval Date-
Regulation-
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Related

1
Clinical Trials associated with AAV1-gamma-sarcoglycan vector(Viromed, Inc.)
NCT01344798
/ CompletedPhase 1IIT
Phase I Clinical Study of AAV1-gamma-sarcoglycan Gene Therapy for Limb Girdle Muscular Dystrophy Type 2C
100 Clinical Results associated with AAV1-gamma-sarcoglycan vector(Viromed, Inc.)
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100 Translational Medicine associated with AAV1-gamma-sarcoglycan vector(Viromed, Inc.)
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100 Patents (Medical) associated with AAV1-gamma-sarcoglycan vector(Viromed, Inc.)
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100 Deals associated with AAV1-gamma-sarcoglycan vector(Viromed, Inc.)
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R&D Status

10 top R&D records.
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IndicationHighest PhaseCountry/LocationOrganizationDate
Limb-Girdle Muscular Dystrophy, Type 2CPhase 1
FR
GenethonGenethon
01 Nov 2006
Muscular Dystrophies, Limb-GirdlePhase 1-
Viromed, Inc.Viromed, Inc.
-
Muscular Dystrophies, Limb-GirdlePhase 1-
GenethonGenethon
-
SarcoglycanopathiesPreclinical
US
GenethonGenethon
-
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Clinical Result

Indication
Phase
Evaluation
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Study
Phase
PopulationAnalyzed EnrollmentGroupResultsEvaluationPublication Date
ASGCT2011
Phase 1
Limb-Girdle Muscular Dystrophy, Type 2C
del525T homozygous mutation on the γSGC gene | absence of γSGC immunostaining in muscle biopsy
9
AAV1 - γ-Sarcoglycan Gene Therapy
mplpmavqck(ksinhxzwph) = one developed a cytotoxic response again AAV1 capsid dujecxhdwl (rrkriicrus )
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Positive
01 May 2011
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Clinical Trial

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Approval

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Regulation

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