Last update 25 Oct 2025

AVXS-301

Overview

Basic Info

Drug Type
AAV based gene therapy
Synonyms
AAV9-SOD1-shRNA (AveXis), AVXS-301
Target
SOD1
Action
inhibitors
Mechanism
SOD1 gene inhibitors
Therapeutic Areas
Nervous System DiseasesEndocrinology and Metabolic DiseaseOther Diseases
Active Indication-
Inactive Indication
Amyotrophic Lateral SclerosisMotor Neuron Disease
Originator Organization
Modalis Therapeutics Corp.Modalis Therapeutics Corp.
Active Organization-
Inactive Organization
Novartis Gene Therapies, Inc.Novartis Gene Therapies, Inc.
Modalis Therapeutics Corp.Modalis Therapeutics Corp.
License Organization-
Drug Highest PhaseDiscontinuedDiscovery
First Approval Date-
Regulation-

Related

100 Clinical Results associated with AVXS-301
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100 Translational Medicine associated with AVXS-301
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100 Patents (Medical) associated with AVXS-301
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100 Deals associated with AVXS-301
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R&D Status

10 top R&D records.
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IndicationHighest PhaseCountry/LocationOrganizationDate
Amyotrophic Lateral SclerosisDiscovery
United States
Novartis Gene Therapies, Inc.Novartis Gene Therapies, Inc.
-
Motor Neuron DiseaseDiscovery
United States
Modalis Therapeutics Corp.Modalis Therapeutics Corp.
-
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Clinical Result

Indication
Phase
Evaluation
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Study
Phase
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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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