Last update 15 Apr 2026

AAV-HTTamiRNA

Overview

Basic Info

Drug Type
miRNA, CRISPR/Cas, AAV based gene therapy
Synonyms
AAV HTTamiRNA, AAVHTTamiRNA
Target
HTT x SOD1
Action
inhibitors, modulators
Mechanism
Huntingtin inhibitors, SOD1 modulators(Superoxide dismutase modulators)
Therapeutic Areas
Nervous System DiseasesCongenital DisordersEndocrinology and Metabolic Disease+ [1]
Active Indication
Amyotrophic Lateral SclerosisHuntington Disease
Inactive Indication-
Originator Organization
Sanofi UK
Active Organization
Arbor Biotechnologies, Inc.Arbor Biotechnologies, Inc.
Sanofi UK
Inactive Organization-
License Organization-
Drug Highest PhasePreclinical
First Approval Date-
Regulation-

Structure/Sequence

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Related

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

10 top R&D records.
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IndicationHighest PhaseCountry/LocationOrganizationDate
Amyotrophic Lateral SclerosisPreclinical
United States
Arbor Biotechnologies, Inc.Arbor Biotechnologies, Inc.
13 May 2025
Huntington DiseasePreclinical
United Kingdom
Sanofi UK
08 May 2024
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Clinical Result

Indication
Phase
Evaluation
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Study
Phase
PopulationAnalyzed EnrollmentGroupResultsEvaluationPublication Date

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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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