Last update 31 Jul 2025

AAV9-hCLN7 (Elpida Therapeutics)

Overview

Basic Info

Drug Type
AAV based gene therapy
Synonyms
Adeno-associated viral vector serotype 9 encoding a codon-optimized human ceroid neuronal lipofuscinosis type 7 (CLN7) transgene
Target
MFSD8
Action
modulators
Mechanism
MFSD8 modulators(Major facilitator superfamily domain-containing protein 8 modulators)
Therapeutic Areas
Nervous System DiseasesCongenital DisordersEndocrinology and Metabolic Disease+ [1]
Active Indication
Neuronal Ceroid-Lipofuscinoses
Inactive Indication-
Originator Organization
Elpida TherapeuticsElpida Therapeutics
Active Organization
Elpida TherapeuticsElpida Therapeutics
Inactive Organization-
License Organization-
Drug Highest PhasePreclinical
First Approval Date-
RegulationOrphan Drug (United States)

Related

100 Clinical Results associated with AAV9-hCLN7 (Elpida Therapeutics)
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100 Translational Medicine associated with AAV9-hCLN7 (Elpida Therapeutics)
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100 Patents (Medical) associated with AAV9-hCLN7 (Elpida Therapeutics)
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100 Deals associated with AAV9-hCLN7 (Elpida Therapeutics)
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R&D Status

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IndicationHighest PhaseCountry/LocationOrganizationDate
Neuronal Ceroid-LipofuscinosesPreclinical
United States
Elpida TherapeuticsElpida Therapeutics
29 Oct 2024
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Clinical Result

Indication
Phase
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Approval

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Regulation

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